Inside Pediatrics Magazine Fall/Winter 2025

PEDIATRICS

Fall/Winter 2025

Lau’s V i s i on NEW PHYSICIAN-IN-CHIEF OUTLINES FUTURE GOALS

A t Children’s of Alabama, we strive to provide the best care for all our patients. And while most of them come from Alabama and neighboring states, much of the work we do influences not just our region but the entire world of medicine. There’s no question that we lead the way in many areas. In this issue of Inside Pediatrics, we’ll show you how. Leading the way often begins with great leadership. This year, we hired a new physician-in-chief, Yung Lau, M.D. He’s served Children’s in the Division of Pediatric Cardiology for three decades, including in leadership roles, and now he’ll lead all divisions in the University of Alabama at Birmingham (UAB) Department of Pediatrics. His goal is to build on the existing foundation of success. Our ability to solve problems also contributes to our influence. Endocrinologist Ambika Ashraf, M.D., exemplified this when she helped develop a framework and a new tool for clinicians to use when determining how to manage hypertriglyceridemia. Likewise, our pulmonology team addressed a challenging situation by starting a new initiative to help asthma patients in an underserved part of our state. We also do leading-edge research that has the potential to change standards of care. Our neonatology team is involved in

Tom Shufflebarger, President and CEO

multiple research efforts designed to address bronchopulmonary dysplasia, or BPD.

In some cases, we’re among just a few hospitals in the nation offering certain treatments or procedures—for example, gene therapy for patients with Duchenne muscular dystrophy (DMD). From the programs we develop to the procedures we offer, Children’s of Alabama is making a difference in all the lives we touch and many more.

Inside this issue:

ADMINISTRATION Tom Shufflebarger, President and CEO Chandler Bibb, Chief Development Officer Garland Stansell, Chief Communications Officer

Division Rounds Quick briefs and news from around the hospital 2 6 10 12

EDITORIAL Conan Gasque, Editor Amy Dabbs

Using Mitochondrial Genetics to Predict BPD Research may allow for early intervention and offer insights

DESIGN Becca Hodges, Lead

New Ways to Manage High Triglycerides in Children Children’s endocrinologist helps develop a framework and tool

Scott LeBlanc Dana Stuckey

PHOTOGRAPY Maurice Adams, Cover Eric Gray Denise McGill CONTRIBUTORS Debra L. Gordon, MS Sarah Handzel, BSN, RN

New Gene Therapy for Patients with DMD Treatment offers hope for a longer, better life

Rhonda Lee Lother Maureen Salamon Judith Thomason Heather Watts

DIGITAL CONTENT Andre Green

MEDICAL LEADERSHIP Yung Lau, M.D. Katharine Reynolds Ireland Chair of Pediatrics Chair, UAB Department of Pediatrics Physician-in-Chief, Children’s of Alabama Mike Chen, M.D., MBA Joseph M. Farley Chair in Pediatric Surgery University of Alabama at Birmingham Chief of Pediatric Surgery & Surgeon-in-Chief Children’s of Alabama Sonya Pritchard, M.D. Professor Chief of Service, Child & Adolescent Psychiatry Children’s of Alabama

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Bringing Asthma Care Closer to Home New initiative aims to help patients in underserved region

Lau’s Vision New Physician-in-Chief outlines future goals

Recognitions News, honors, awards and leadership

For questions or additional information or to share feedback, please contact us at InsidePediatrics@ChildrensAL.org . An online version of the magazine is available at ChildrensAL.org/InsidePediatrics.

DIVISION ROUNDS

For a closer look at these stories and more, visit InsidePeds.org

UROLOGY

A new protocol for kidney tests in spina bifida patients

hydronephrosis had only about a 25% sensitivity for identifying children with signs of chronic kidney disease in the UMPIRE study and 24% in the NSBPR cohort. That means kidney damage in three out of four children was going undetected. The poor sensitivity held even when researchers looked only at severe hydronephrosis, which had an even worse sensitivity rate—just 6% to 11%. “The renal ultrasound by itself wasn’t all that good,” Joseph said, “but that didn’t surprise us.”

A new study from Children’s of Alabama urologists Stacy Tanaka, M.D., and David Joseph, M.D.—along with other kidney experts from around the country—shows that ultrasound alone is not enough to assess kidney health in spina bifida patients. This is important because people with spina bifida tend to develop end-stage renal disease up to 20 years earlier than the general population, so keeping a close watch on their kidney health from a young age is crucial. Current guidelines from the Spina Bifida Association (SBA) recommend annual screening with ultrasound to look for hydronephrosis—a condition in which the urine backs up into one or more kidneys—as a sign of kidney function and blood tests, such as serum creatinine, to measure overall kidney health. But with kids, Tanaka said, “the practice pattern was that a lot of people were only doing renal ultrasound.” “We basically use ultrasonography as a reflection of renal function,” Joseph added, but few, if any, studies had assessed its accuracy in determining renal function. In the study, investigators used data from the National Spina Bifida Patient Registry (NSBPR) and UMPIRE, a urologic protocol, to manage and preserve initial renal function in young children with spina bifida. The two registries include data on 2,500 children ages 1–18 with myelomeningocele, the most severe form of spina bifida. All had undergone an ultrasound and blood test within six months to determine estimated glomerular filtration rate (eGFR), a marker of kidney health. The results were striking: Ultrasound-based detection of

Ultrasound-based detection of hydronephrosis has only about a 25% sensitivity 25 %

Children with spina bifida have undetected kidney damage 3 out of 4

The findings challenge current practice and suggest that blood tests measuring kidney function should be routinely performed alongside ultrasound, not just when ultrasound results look concerning, as some clinicians practice. “The ultrasound is very helpful and important,” Joseph said, “but you need to recognize that it may not be telling you about renal function or injury to the kidney.” The findings have already changed practice at Children’s, where all kids with spina bifida now receive both tests during kidney health screening.

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GASTROENTEROLOGY

New technology eases management of liver disease

Reducing hypertension numbers in children–and adults Successfully stemming the rising rates of cardiovascular disease in adults needs to start in childhood. But with mounting numbers of children and adolescents developing high blood pressure—a trend driven largely by skyrocketing obesity rates—this objective is getting harder to achieve. The Pediatric Hypertension Program at Children’s of Alabama, with its steady growth, seeks to break the cycle. The Hypertension Clinic, which operates three half-days each week, now sees about 45 patients weekly, a tenfold increase from 14 years ago, says nephrologist Daniel Feig, M.D., Ph.D., who was recruited in 2011 to oversee the clinic’s development and expansion. With obesity in children steadily rising, more young patients are coming to Children’s of Alabama with a form of fatty liver disease that can greatly imperil their health. But determining the progression of liver disease can be a thorny process. To smooth that path, Children’s recently invested in an increasingly popular technology called FibroScan, helping UAB pediatric physicians to deftly and comprehensively manage children’s care. Using a technique known as transient elastography, FibroScan was the first FDA-approved device of its kind and is considered an aid to managing liver disease. Quick, noninvasive and painless, it uses an enhanced form of ultrasound to send vibrations into the liver to measure its stiffness, which typically indicates fibrosis or scarring. By assessing the severity of scarring—and the potential for cirrhosis—FibroScan can help diagnose or monitor the progression of various liver conditions. These range from less-common cystic fibrosis-associated liver disease to more prevalent autoimmune liver diseases and metabolic dysfunction-associated steatotic liver disease (MASLD). About one-third of the patients in the Children’s Hepatology Clinic, which serves about 500 ongoing patients each year, have NEPHROLOGY

Image courtesy of Echosens

suspected or confirmed fatty liver disease. “We will be using this for almost every patient with confirmed fatty liver disease, so we can measure the baseline stiffness of the liver when they first see us,” said David Willcutts, M.D., a transplant hepatologist at Children’s. FibroScan is an alternative to invasive liver biopsies and other forms of elastography that require a separate radiology appointment. A FibroScan exam takes just minutes, offering little disruption for young patients and faster treatment decisions for physicians. The new equipment arrived this summer.

High blood pressure—defined in adults and children 13 years and older as a reading of 130/80 mm Hg or higher— is relatively unusual in healthy young patients, affecting 2–3% of typical children and adolescents. But children with obesity—who account for nearly 20% of all Americans under 18—have a 20–30% rate of hypertension, Feig says. “There’s a fairly large number of kids with

20 - 30 % of children with

obesity have hypertension

high blood pressure, and one of the major concerns is how we can impact the long-term epidemic that results in cardiovascular disease in adults,” said Feig, who’s also the director of the Division of Pediatric Nephrology at Children’s. “The only way we can get this under control is by prevention,” he added. “If we can make an impact in children and adolescents, we can push back cardiovascular disease in adults.” Ultimately, the team is trying to prevent both short- and long-term health implications resulting from hypertension, including heart thickening, retinal damage and even impairments in cognitive function. It’s a daunting task, Feig acknowledges. “Anything we can do to reduce the numbers right now has a domino effect that reduces long-term target organ damage and long-term cardiovascular risk,” he said. “We’re not at a point where we can reverse the trajectory in 70,000 undiagnosed kids in Alabama, but we can positively impact a good number of kids, improve their health and quality of life, and gain the opportunity to gradually access more and more of them.”

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Inside Pediatrics | Children’s of Alabama

DIVISION ROUNDS (cont.)

BEHAVIORAL HEALTH

Children’s expands mental health program into schools As the mental health crisis continues across the country, children and teens are in greater need of mental health services than ever before. In many cases, the first person to recognize the child’s need is a teacher. But the teacher may not always know how to help. That’s why the Children’s of Alabama behavioral health team recently expanded its PATHS program to school personnel. PATHS, or Pediatric Access to Telemental Health Services, has been around since 2019. Children’s established it—with support and funding from the Alabama Department of Mental Health (ADMH)—in response to the need for more mental health services, especially in rural areas. The initial goal was to help primary care providers understand how to help patients who present with mental health concerns. Through the program, Children’s mental health professionals offer consults or education for providers or even telehealth appointments for their patients. PATHS has since expanded into urban areas, and now, thanks to the extension of a Health Resources and Services Administration (HRSA) grant, Children’s is offering support to school systems across the state. “This is an important step for our program,” PATHS director Margo Harwell said. “Because they see students daily, school personnel play a critical role in identifying early signs of mental health concerns in students.”

ORTHOPEDICS

How dogs are transforming pediatric orthopedic procedures What goes in must come out—including the metal pins used to hold bones together while fractures heal. “It probably takes 15 seconds to remove three pins,” Children’s of Alabama pediatric orthopedist Michael Conklin, M.D., said. “But, of course, kids are very scared about that.” Enter Shelby. The 50-pound standard poodle is trained to sit on the examining table and cuddle with children while Conklin grasps the pins with a tool resembling a needle-nosed plier and pulls them out. Basically, Shelby serves as a distraction, he said. “We tell the child to pet the dog and look toward the dog and not look at me on the other side of them, not worry about what I’m doing.” For about two-thirds of patients, it works. And it even helps the ones who “freak out no matter what,” Conklin said. “It seems as if they return back to their baseline calm quicker after the procedure.” Shelby, and her brother, Foster, are part of the Pups Unleashing Patient Smiles (PUPS) program, which is one of three branches of Children’s of Alabama’s animal-assisted program, PetsRX. Another branch involves longtime Children’s partner, Hand-in-Paw, which provides therapy dogs at Children’s of Alabama’s main hospital to provide comfort and distraction. The third is a hospital based medical dog program which includes golden retrievers Wanda and Sydney to assist with scary or painful procedures. Meanwhile, suspected victims of child abuse—who are served by the Children’s Hospital Intervention and Prevention Services Center (CHIPS)—are assisted by dogs from the Help Empower Restore Overcome (HERO) Program with the Alabama Office of Prosecution Services. Soon, Shelby and Foster may be part of the scientific literature. Conklin and his team are conducting a randomized trial to evaluate the dogs’ effectiveness, comparing outcomes between patients who receive the therapy dog intervention and those who receive standard care. The goal is to, hopefully, show positive data that will pave the way for broader adoption of such programs.

Leaders began their efforts in the summer of 2024, meeting with mental health coordinators in school systems across Alabama to discuss what topics might need to be covered in their respective districts. Once those needs were identified, the PATHS team set up in-person, virtual or hybrid training sessions. PATHS leaders have offered training on various mental health topics, including anxiety, behavior management, depression, trauma and bullying. They’ve also taught educators how to identify the red flags of mental health concerns among students. The sessions are opportunities for discussion among school staff and Children’s mental health experts. Ultimately, PATHS leaders hope to help educators understand how to handle mental health issues in students as they arise.

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HEMATOLOGY & ONCOLOGY

Aye appointed director of Developmental Therapeutics As the new director of Developmental Therapeutics,

Inside Pediatrics provides information, news and the latest updates from departments and divisions at Children’s of Alabama. Articles and features cover the current developments and discoveries related to pediatric medical research, clinical trials and treatments happening today. Inside Pediatrics helps keep you connected and up-to-date on the latest breakthroughs and medical advances at Children’s of Alabama.

Jamie Aye, M.D., aims to improve patient outcomes through the development of new clinical trials. Aye, an associate professor at UAB, was appointed director of the program in July after serving as associate director since 2023. In that role, she worked toward the mission of discovering, developing and delivering more effective and less toxic therapies for children

with cancer and blood disorders through translational research and early-phase clinical trials. In her new role, she will focus on research collaborations to advance clinical trials. “My vision is to improve the outcomes for patients with childhood cancer and blood disorders by strengthening collaborations between researchers locally at UAB and with researchers nationally,” Aye said. “Through these collaborations and in partnership with the Pediatric Clinical Trials Office, my goal is to develop cutting-edge, investigator-initiated trials here at Children’s of Alabama.” The Developmental Therapeutics Program is part of the Division of Hematology, Oncology, and Blood & Marrow Transplantation at UAB and Children’s. Its purpose is to improve the cure rate of pediatric cancers and blood disorders while also decreasing immediate and long-term side effects experienced by children undergoing treatment. Patients referred to the program typically have recurrent or refractory malignancies that have failed standard therapies. Other patients may be diagnosed with rare tumors for which no effective therapies have been established. DIVISION ROUNDS is a brief roundup of the innovative work Children’s of Alabama’s medical experts are doing in various divisions around the hospital. It showcases how we’re improving patient care and influencing the world of medicine. For more details and to find similar stories, please visit InsidePeds.org .

CATEGORIES

BEHAVIORAL HEALTH

CARDIOLOGY

ENDOCRINOLOGY

GASTROENTEROLOGY

HEMATOLOGY & ONCOLOGY

NEONATOLOGY

NEPHROLOGY

NEUROLOGY & NEUROSURGERY

ORTHOPEDICS

PULMONOLOGY

UROLOGY

UPCOMING EVENTS

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NEONATOLOGY

Using Mitochondrial Genetics to Predict BPD

SOLVING THE PUZZLE FOR EARLY INTERVENTION AND INSIGHT INTO RACIAL DISPARITIES

B ronchopulmonary dysplasia (BPD), a chronic lung condition affecting some extremely preterm infants, continues to be a significant clinical challenge in neonatology. While often lifesaving, supplemental oxygen can be a key contributor to long-term pulmonary complications in this vulnerable population. At Children’s of Alabama and the University of Alabama at Birmingham (UAB), researchers are exploring how mitochondrial function may hold the key to understanding and preventing BPD. Jegen Kandasamy, M.D., an associate professor in the Division of Neonatology at UAB, leads a multidisciplinary team supported by a research grant dedicated to studying mitochondrial dysfunction in BPD. The research centers on individual differences in how mitochondrial DNA (mtDNA) haplogroups—genetic variations inherited maternally and varying by ethnicity—may influence an infant’s susceptibility to lung injury from oxygen exposure, particularly hyperoxia. “Hyperoxia is a double-edged sword,” Kandasamy said. “It’s essential for survival, yet it introduces oxidative stress that preterm lungs are poorly equipped to handle. Our research is aimed at understanding how mitochondrial genetics impact that response.” Using collected blood samples and clinical data from preterm infants, Kandasamy’s team is working to identify mtDNA haplogroups associated with higher BPD risk. The goal is to develop precise, genetically informed risk profiles that allow for early intervention. Hopefully, this will improve outcomes while addressing racial disparities in BPD prevalence and severity.

Jegen Kandasamy, M.D.

“Understanding how mitochondrial genetics intersect with environmental exposures can help us identify at-risk infants earlier and intervene more effectively.”

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Inside Pediatrics | Children’s of Alabama

The team is also studying mitophagy, the elimination of damaged mitochondria through autophagy, and its role in lung development. Emerging evidence suggests that impaired mitophagy contributes to persistent mitochondrial dysfunction, exacerbating lung injury in preterm infants. As a result of this new evidence, the group is also evaluating the potential of thyroid hormone supplementation as a therapeutic strategy to restore mitochondrial function and mitigate lung damage. By integrating clinical data with mouse models, the UAB team is uniquely positioned to investigate both the mechanistic underpinnings of BPD and potential interventions. The collaborative effort spans neonatology, mitochondrial biology and pediatric pulmonology, creating a comprehensive research environment. “Our ultimate aim is to shift the paradigm from reactive to predictive personalized neonatal care,” Kandasamy said. “Understanding how mitochondrial genetics intersect with environmental exposures can help us identify at-risk infants earlier and intervene more effectively.” ●

Chest X-ray of a child with bronchopulmonary dysplasia

An especially promising area of research is platelet bioenergetics. By measuring how platelets utilize mitochondrial energy, the researchers hope to identify specific biomarkers that reflect systemic mitochondrial health and may help predict BPD risk. “Platelets are easy to access and give us a real-time snapshot of mitochondrial function without invasive procedures,” Kandasamy noted.

Children’s of Alabama’s work on Bronchopulmonary Dysplasia

to months before diagnosis of that disease. Viral Jain, M.D. has done extensive work related to chorioamnionitis, and one of his studies suggests that infants born early due to chorioamnionitis have chronic lung damage, including

BPD is a major point of focus for the Children’s neonatology team. In addition to Kandasamy’s work on mitochondrial genetics, the team has published other studies designed to predict the onset of the condition, including research done by Namasivayam Ambalavanan, M.D., which shows

BPD. And as you saw in the last issue of Inside Pediatrics, Colm Travers, M.D., developed a device that uses sound waves to improve lung testing in prematurely born babies. The team is also working to develop ways to treat preterm infants, including those who have BPD. For example, a study led by Waldemar Carlo, M.D., shows that preterm infants receiving caffeine treatment were apnea free sooner than those who did not.

that physiologic data can predict unfavorable respiratory outcomes, such as BPD, in extremely preterm infants. Kent Willis, M.D., also recently published a study that concludes that the gut composition of fungi in the second week of life predicts the later development of BPD, weeks

For more on the work the Children’s neonatology team is doing related to BPD, search “neonatology" at InsidePeds.org.

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Discovery

happens

When people with extraordinary talent and passion are given the technology, the facilities and the support, they achieve great things. The discoveries and innovations happening today will help shape the future of treatments and lead to cures. And it benefits not only the patients and families who come to Children’s of Alabama, but people across the country and around the world for years to come.

Learn more at ChildrensAL.org

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Inside Pediatrics | Children’s of Alabama

Sharing Solutions CHILDREN’S ENDOCRINOLOGIST HELPS DEVELOP NEW TOOL AND FRAMEWORK TO MANAGE HIGH TRIGLYCERIDES IN CHILDREN

I n recent years, endocrinologists at Children’s of Alabama have seen a drastic increase in the number of young patients with severe hypertriglyceridemia—extremely elevated triglyceride levels that sometimes exceed 1,000 mg/dL, posing serious health risks if left untreated. This growing trend reflects a broader national concern: hypertriglyceridemia affects an estimated 10-20% of youth in the U.S., with prevalence reaching as high as 40–60% among children and adolescents with obesity.

of youth with obesity are affected by hypertriglyceridemia

% 40 - 60

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ENDOCRINOLOGY

But for many pediatricians and nurse practitioners, figuring out the best approach to manage this condition can be confusing. Recognizing the need for better clarity in diagnosing and managing these children and adolescents, the Division of Pediatric Endocrinology at Children’s of Alabama and the University of Alabama at Birmingham (UAB) spearheaded a pair of major research efforts. Ambika Ashraf, M.D., director of the division, teamed up with mentee Charles Gagnon, M.D., now a pediatric resident at Boston Children’s Hospital, to write a much-needed review in Current Atherosclerosis Reports that aims to provide a clear, practical framework clinicians can use in everyday care. The article breaks down the various causes of high triglycerides in children, highlights when to worry, and outlines treatment strategies that range from lifestyle changes to medications. It also explains when clinicians should consider emerging therapies and what to look for to prevent serious complications such as pancreatitis. “This work reflects our commitment to bridging academic knowledge with clinical practice, making a difference where it matters most: at the bedside,” said Ashraf, also the Ralph Frohsin Endowed Chair in Pediatric Endocrinology at UAB. “With a very high percentage of children affected by obesity, we are seeing a large number of pediatric patients with elevated triglyceride levels in our clinics.” Ashraf also partnered with leading experts across North America to develop the first validated scoring tool in the region for familial chylomicronemia syndrome (FCS), a rare genetic disorder often implicated in severe hypertriglyceridemia. But Ashraf and her colleagues recognize that that some of these children may instead have an acquired condition called multifactorial chylomicronemia syndrome (MCS), which requires a different treatment approach.

Scan to download and use the NAFCS score calculation tool.

1 year or older with severe hypertriglyceridemia. It incorporates factors such as body mass index (BMI), history of pancreatitis, triglyceride levels, apolipoprotein B levels and the presence of secondary contributors such as diabetes, medications or hormonal disorders. The results of this collaborative effort were recently published in the Journal of Clinical Lipidology, marking a milestone in pediatric lipid care. The NAFCS score is not only a practical tool for frontline clinicians, Ashraf says, but also a prime example of how academic expertise can translate into improved patient outcomes. In addition, it may assist in confirming a clinical diagnosis of FCS in cases with inconclusive genetic testing. “This has been a growing need,” Ashraf said. “Everyone on the panel who helped develop this tool thought this will help shape the current and future management of patients with FCS in the United States and Canada.” The Division of Pediatric Endocrinology’s involvement in this research highlights UAB’s national role in shaping pediatric lipid care, says Ashraf, who also serves as the director of the Pediatric Lipid Clinic at Children’s. In addition, faculty members actively participate in national working groups dedicated to lipid disorders, rare genetic diseases and pediatric obesity. “We hope this new tool empowers more accurate diagnoses and more personalized, effective care for children struggling with complex lipid disorders,” Ashraf said. “For families, it offers hope, and for clinicians, it offers clarity.” •

The new scoring tool, known as the North American FCS Score (NAFCS), is designed to help providers distinguish between FCS and MCS in patients aged

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Inside Pediatrics | Children’s of Alabama

NEUROLOGY

New Gene Therapy Offers Hope for Patients with DMD I n January 2025, Children’s of Alabama, for the first time, treated a patient with Duchenne muscular dystrophy (DMD) using a new gene therapy offered by only a few academic hospital facilities in the nation. The milestone followed a lengthy approval process and marked a new opportunity for patient success and scientific progress. Though not a cure, the treatment represents the hope of a longer life for these patients. For researchers, it will contribute to greater learning about the potential of this new treatment.

While it is considered a rare disease, DMD is the most common form of muscular dystrophy, affecting one in every 5,000 males born in the United States. Patients experience progressive muscle degeneration, starting with proximal muscles and expanding to the limbs over time. They have trouble with many physical activities such as jumping, running and walking, and lose the ability to walk over time. The disease is fatal, and most patients don’t live past their late 20s. DMD has no cure; treatment focuses on extending the patient’s life by slowing down its progression. According to the Muscular Dystrophy Association, symptoms of DMD can begin as early as ages 2–3 years, but in Alabama, where DMD is not yet part of newborn screening, many boys are not diagnosed until ages 4–6. That, says Children’s neuromuscular nurse practitioner Samantha Weaver, DNP, CRNP, is when patients begin to experience a steady decline.

Muscle atrophy

Healthy muscle

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Treatment

“Now that we’re starting to get these really breakthrough therapies, they’re fulfilling on the promise that we all were searching for, which is that we could get closer to making this disease really better.”

Since the 1990s, physicians have prolonged the lives of patients with DMD using corticosteroids, whose anti inflammatory properties can slow down the disease’s progression by about three years. Gene therapy, however, represents a new treatment aimed at restoring the function of the causative gene, dystrophin. The U.S. Food and Drug Administration originally approved it in 2023 for use in patients ages 4–5. In 2024, the agency extended that approval to all patients 4 years and older. In this treatment, the transgene (a micro-dystrophin synthetic gene) is packaged within a viral capsid—a virus not intended to harm the patient that can hold the genetic material. In essence, physicians are “giving back the missing genetic information to the muscle tissue,” Children’s neurologist Michael Lopez, M.D., Ph.D., said. Unlike any other option, he noted, gene therapy treats the root cause of DMD. “Now that we’re starting to get these really breakthrough therapies, they’re fulfilling on the promise that we all were searching for, which is that we could get closer to making this disease really better,” he said.

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Inside Pediatrics | Children’s of Alabama

For the patient, improvements don’t happen overnight. That’s not how gene therapy works, Lopez says. “What we hope is that over many years, we’ll see a slow progression of the disease that is beyond what we would get with just treatment with corticosteroids alone,” he explained. “And I think that added benefit is something that’s going to be more of a long-term improvement.”

Finding the Right Fit

Gene therapy, however, is not the right fit for every patient. To determine candidates, Children’s looks at age, underlying disease and disease progression, Weaver said. They also consider the patient’s overall health and risk for infectious diseases. “It’s an extensive process,” she explained. With all gene therapies, safety must be prioritized. The treatment can produce a significant immune response that can even prove life-threatening to patients with more advanced stages of the disease. Liver injury is also a major concern. Thus, Weaver says the team must ensure the patient has no antibodies that will reject the virus. “These are important steps to make sure the patient will have the best outcome,” she said.

So, what can the parents of

each patient treated with gene therapy hope to see? Ideally, in the short-term, their

child will be more active. “I hope that our children can have more of

a shot at more play and more jumping and more climbing and all of those things in the future,” said Erin McLeod, M.D., a pediatric neuromuscular neurologist at Children’s. In the long-term, the hope is that they’ll have a longer life. Evidence supports the treatment’s efficacy. The clinical trials show that gene therapy is being delivered to patient’s muscles, and while the motor assessments haven’t shown clear evidence of clinically observable benefits, the data has trended toward improvement. Lopez says in other, more recent studies, treated patients are starting to show improvements compared to those not receiving gene therapy. “The MRIs of the muscles themselves look a little bit healthier in some of these patients,” he said. “There’s less evidence of disease in that.”

Because of these considerations, only a small percentage of patients are ideal for the treatment.

Why Offer It at Children’s?

In Alabama, Children’s is the only hospital that offers gene therapy for patients with DMD. Making it available made sense—the hospital already treats spinal muscular atrophy (SMA) patients with gene therapy. Brad Troxler, M.D., and Shelley Coskery, CRNP, led the way on that, Lopez said, and “built in a lot of the infrastructure that we needed to be able to start doing gene therapies.” “That really has put us out in front of the field with the experience to deliver these high-cost and novel leading-edge treatments,” he added.

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Challenges

Cost was one of many challenges for the team as they sought approval to implement this multimillion-dollar therapy. To that end, they involved hospital administration and a pharmacoeconomics committee in the process. But, as Weaver pointed out, the process involved many more steps, including determining who would write a protocol to ensure patient safety. They also had to build a larger team, which ultimately included hepatologists, pulmonologists, cardiologists, physical therapists and social workers. Obstacles persist, even as Children’s offers the treatment. “A high cost remains a big challenge,” Lopez said. “So we’ve been fortunate to be able to provide these treatments because we’ve gotten support from the insurers, so far.” But not every insurer is the same, he noted, and some may be slow to cover or even decline to cover the treatment.

“This first approved treatment for Duchenne that is a gene therapy is just the beginning, and there are going to be more down the road,” Lopez added. “There are certainly some that are in clinical trials now. So, I think we’re right to be optimistic in that we’re starting to really push the treatment of Duchenne in a way that’s going to give us lots of options that weren’t there before.” And as Children’s continues to offer the treatment, they’ll contribute to the scientific community’s information on its effectiveness, which means the team is paving the way toward greater success for the broader population of patients with DMD. “I think everyone who is familiar with it at this point knows it is not a cure. But it is supposed to significantly slow the disease, and we are still gathering and gaining more data to that,” Weaver said. “So we’re very excited to be part of that process.” •

What the Future Holds

So far, Children’s has dosed only one patient— out of roughly 100 that it follows—with the new gene therapy. While few will be candidates for the treatment, the team hopes to dose more in the future, as long as “the risk is appropriate and the benefit is continuing to be demonstrated,” Lopez said. The team also expects more advancements, which may make it possible for others—especially those with more severe cases—to receive the treatment.

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Inside Pediatrics | Children’s of Alabama

PULMONOLOGY

Bringing Asthma Care Closer to Home in Alabama’s Black Belt

NEW INITIATIVE TO HELP ASTHMA PATIENTS IN UNDERSERVED REGION

D allas, Marengo, Perry and Wilcox counties, part of the Black Belt (so named for its rich, dark soil), are four of the poorest counties in Alabama. They also have some of the highest rates of childhood asthma— nearly 12% compared to the state’s 8%—and are severely underserved when it comes to medical care. Two of the counties don’t even have a pediatrician.

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“We realized these kids weren’t getting to us for help. But Medicaid data showed a high number of asthma-related claims from the area. So we knew there was a gap.”

The initiative began when Children’s and UAB infectious disease specialist Claudette Poole, M.D., spent time in the area studying water sanitation and parasites. She kept hearing about an asthma crisis and recruited Virella-Lowell and Magruder. The three applied for and received a Health Resources and Services Administration (HRSA) grant, which provides salary support for the core team and local community and health care partners. It also helps fund the virtual continuing medication education (CME) Project ECHO sessions, health fairs and supplies—such as spirometers and educational materials. Magruder and Virella-Lowell stress the community based approach of their efforts rather than having Children’s swoop in for just a few months. “We are really trying to improve the capacity of the community and their understanding of delivering asthma care in their own communities,” said Magruder. That means educating the frontline

Yet just 4% of the more than 5,000 patients seen at Children’s of Alabama’s Specialty Asthma Clinic hail from those areas. “We realized these kids weren’t getting to us for help,” pediatric pulmonologist Isabel L. Virella-Lowell, M.D., said. “But Medicaid data showed a high number of asthma-related claims from the area. So we knew there was a gap.” One reason is distance, with families having to drive up to three hours to reach Birmingham. Thus, many children receive care only during asthma flare-ups at urgent care clinics or emergency rooms rather than ongoing, preventive treatment, said Children’s and University of Alabama at Birmingham (UAB) Pediatric Asthma Program Director Teresa G. Magruder, M.D. Without a primary care physician overseeing their child’s asthma, families find themselves caught in a cycle of crisis-driven care. So instead of hoping kids will come to Birmingham, Virella-Lowell and Magruder are bringing their expertise to the Black Belt. Their mission: improve those dismal asthma statistics by engaging the community at a grassroots level.

Isabel Virella-Lowell, M.D. (left) and Teresa Magruder, M.D. (right)

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Inside Pediatrics | Children’s of Alabama

people caring for children, including school nurses, teachers, daycare workers and parents, while providing access to subspecialty care for the severe high-risk patients.

“If the community doesn’t buy in, if they are not engaged, if they’re not supportive, then there’s a limited amount of good we can do,” Virella-Lowell said.

“There’s some fatigue in these communities from programs that come and go,” Magruder added. “We know it takes time to build trust.”

The two doctors are also partnering with local physicians at Selma Pediatrics and Whitfield Regional Hospital in Demopolis. They hope to open a monthly clinic in 2026 in a space provided by Selma Pediatrics, so families don’t have to travel so far for specialty care. They are also educating clinicians and others who see children with asthma through the aforementioned CME approach called Project ECHO (which stands for Extension of Community Healthcare Outcomes), an interactive program that helps clinicians address their own cases. “It’s incredibly important that local physicians are comfortable managing asthma. And asthma care has changed a lot through the years,” said Virella-Lowell, who noted the recent release of new guidelines for diagnosing and treating the disease. The program, which is virtual, is available to any clinician throughout the state and beyond who’s interested in maintaining their expertise in pediatric asthma. The Alabama State Asthma Coalition, a statewide group with a diverse mix of experts, including environmental experts and respiratory educators, is also playing a role. The coalition helped train the first group of community health workers and hopes to continue that work. Given that asthma affects one out of 10 children, it must be managed locally, Virella-Lowell said. “There’s no way that we will ever be able to manage all the really sick asthmatics here at Children’s. So it’s incredibly important that local pediatricians and family doctors are comfortable managing asthma.” “Our goal is to raise the level of asthma care across the state,” Magruder said, “not just at Children’s, but everywhere kids need it.” ●

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Inside Pediatrics | Children’s of Alabama

A V ision f or

“I’ve considered it a privilege to be a part of the department as a faculty member for over three decades. Now, it’s an awesome responsibility to carry on this tradition of excellence.” Yung Lau, M.D.

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LEADERSHIP

th e F u t ure

YUNG LAU’S VISION AS NEW PHYSICIAN-IN-CHIEF

A fter serving as interim chair for five months, Yung Lau, M.D., was officially named chair of the University of Alabama at Birmingham (UAB) Department of Pediatrics and physician-in chief at Children’s of Alabama in March 2025. The only real change, Lau says, was that he could now formally begin planning for the future. His vision for the department is expansive— centered on collaboration and faculty support. But he believes the path to those big goals lies in the small things everyone does every day. Lau stepped into the interim chair role in November 2024, following the announcement that then-chair Mitch Cohen, M.D., would be departing at year’s end to join Stanford. “Dr. Cohen led for a decade and helped build this department into a strong and vibrant place,” Lau said. “It has consistently thrived, and I’ve considered it a privilege to be

a part of the department as a faculty member for over three decades. Now, it’s an awesome responsibility to carry on this tradition of excellence.” The role is unique, requiring close collaboration between two major institutions: Children’s of Alabama and the University of Alabama at Birmingham. With more than 30 years of experience across both organizations—including in leadership roles—Lau understands their individual missions and how they intersect.

two institutions. At the time, UAB housed the pediatric cardiac program. But as Children’s leaders planned to build a new hospital building, they wanted to bring the program under their roof. Over the next five years, Lau played a significant role in bringing physicians and other clinical staff to assist in the design of the cardiac intensive care units, operating rooms, catheterization labs, step-down units and cardiovascular perioperative areas. He then worked closely with Children’s leadership on staffing and operations planning. On October 14, 2012, the program moved into the new building—seamlessly. The success of the move laid the foundation for a quantum leap in the ability of the Pediatric and Congenital Heart Center of Alabama to provide state-of the-art care. Today, the program consistently ranks among the top heart centers in the nation.

That understanding came into sharp focus in 2007, when Lau led the UAB group in a major collaborative effort between the

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Inside Pediatrics | Children’s of Alabama

Law Appointed Division Director

Education and research are crucial parts of Lau's strategy, and UAB and Children’s have a history of successful collaboration on both. From an education perspective, Children’s serves as the teaching hospital for the UAB pediatric medicine, surgery, psychiatry, research and residency programs.

of Pediatric Cardiology

Following the promotion of Yung Lau, M.D., to

physician-in-chief at Children’s of Alabama, pediatric cardiologist Mark Law, M.D., was named Lau’s successor as director of the Division of Pediatric Cardiology.

“There’s this deep core of understanding between Children’s and

the department that we are really training the future physicians for the state,” Lau said. And that’s a crucial role in a state that, Lau says, needs more physicians and pediatricians. “Part of our duty here is in our obligation to do that,” he added. On the research side, the two institutions work together to “advance knowledge for the children of Alabama and beyond for the future,” Lau said. This benefits both entities, sometimes leads to advancements and breakthroughs that influence the broader world of medicine, and enhances the reputation of both. Faculty support is another central pillar of Lau’s vision. Since becoming chair, he has met with many faculty members—some he’s long known, others he’s come to know better through these conversations. What stands out most, he says, is their passion and the profound impact they have on children’s lives. His goal is to listen, support and help them succeed.

So far in his new role, Law, a professor in the Department of Pediatrics, has made substantive changes that address current needs and lay a strong foundation for continued growth and success within the division and the Pediatric and Congenital Heart Center of Alabama at Children’s. Widely respected as a leader in pediatric and interventional cardiology as well as adult congenital heart disease, Law has authored or co-authored more than 70 peer-reviewed articles and book chapters. He has also mentored more than 20 postdoctoral fellows and junior faculty, contributing meaningfully to the future of academic medicine. He also serves as medical director of Adult Congenital Interventional Cardiology at UAB Medicine, with a secondary appointment in the Division of Cardiovascular Disease within the Department of Medicine. Prior to his promotion to division director, Law was appointed to the prestigious Lionel M. Bargeron Endowed Chair in Pediatric Cardiology by the Board of Trustees of the University of Alabama.

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Lau also acknowledges the tension between moral obligation and financial reality. “That’s just medicine in America today,” he said. But he’s confident the department can thrive within that framework. “I think both institutions understand that we need to maximize our resources—our people and infrastructure—to provide the best possible care, to train the next generation of pediatricians, and to innovate through research,” he said. Though there are multiple facets to Lau’s vision, everything is centered around the patient.

“The patients in front of us are the cornerstone of everything we do,” he said. “And while our goals may be big, the real progress happens in the small steps we take every day.” “Yes, having a goal is important,” he continued. “But sometimes if we focus only on the goal, we risk losing sight of what’s happening in the moment—and that can distort the work being done on the ground. Sometimes the things that matter most get sidelined in the name of progress.” With a strong focus on the patient—and through strong collaboration and faculty support—

Lau believes UAB and Children’s will continue to deliver exceptional care to every child they serve. •

“And while our goals may be big, the real progress happens in the small steps we take every day.”

“When we collaborate [...] two important things happen: first, children receive better care, now and in the future; second, our faculty experience significantly greater job satisfaction.”

Inside Pediatrics | Children’s of Alabama Inside Pediatrics | Children’s of Alabama

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Recognitions

News, Leadership, Honors, Awards

NEWS

of all Americans. The AAP Council is responsible for reviewing the nominee applications for admittance to the AAP. Kutny Elected to COG Voting Body Steering Committee. Matthew Kutny, M.D., professor in the Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, has been elected to the Children’s Oncology Group (COG) Voting Body Steering Committee. The COG brings together thousands of experts from the U.S., Canada, Australia, New Zealand and Saudia Arabia in the fight against childhood cancer. Ilonze selected for Pediatric Hematology Oncology Sub-board of the American Board of Pediatrics. Chibuzo Ilonze, M.D., assistant professor in the Division of Pediatric Hematology, Oncology and Blood and Marrow Transplantation, was selected to serve on the Sub-board of Pediatric Hematology/Oncology of the American Board of Pediatrics (ABP). He will serve a six-year term beginning in January 2026. Each sub-board of the ABP works with test development staff to develop examination questions as subject matter experts, establish passing standards, and conduct form review. Timmerman elected chair of the Research Coordinator Network for CARRA. Livie Timmerman, clinical research coordinator II for the Division of Pediatric Rheumatology, has been elected to serve a three-year term as chair of the Research Coordinator Network for the Childhood Arthritis and Rheumatology Research Alliance (CARRA). Established in 2002, CARRA is a collaborative research network comprising more than 800 members across more than 120 institutions, dedicated to advancing research in pediatric rheumatic diseases.

leaders. Its mission is to shape the future of academic pediatrics by engaging distinguished child and adolescent health leaders and scholars who represent the diversity of the field. “I look forward to continuing our efforts to increase the pediatric workforce by supporting pathway programs and career development, translating research into policy and practice, increasing health care financing to improve access and address social determinants of health, and engaging youth and communities to improve the health and well-being of all children and adolescents,” Coyne Beasley said. “We will also prioritize working collaboratively to nurture and support leaders and develop innovative and effective approaches to address child and adolescent health challenges and crises.” Salas Named SSPR President-Elect. Ariel Salas, M.D., MSPH, associate professor in the Division of Neonatology, has been named the 2026–2027 Southern Society for Pediatric Research (SSPR) president-elect. The SSPR strives to improve health outcomes for all children through the support of rigorous research, quality improvement and advancing cutting edge clinical care. Kimberlin Elected to Association of American Physicians Council. David Kimberlin, M.D., distinguished professor in the Division of Pediatric Infectious Diseases, has been elected to the Association of American Physicians (AAP) Council. He began a five-year term in May. “I am honored to serve the AAP as council member,” Kimberlin said. “UAB has many esteemed faculty that have been elected as members of the AAP over the years, and I hope to contribute to the advancement of the society’s mission during my years on the council.” The AAP was established in 1885 as an elected society of America’s leading physician-scientists, who are the pinnacle of long-lasting and impactful contributions to improving the health

Corn Masa Fortification Bill Passes. This spring, the Alabama legislature passed the Corn Masa Fortification Bill into law, marking a crucial milestone in Children’s of Alabama’s work to prevent spina bifida. “This is a major win for fortification and is a landmark for Alabama,” Children’s neurosurgeon Jeffrey Blount, M.D., said. Blount, in 2019, co-founded the Global Alliance for the Prevention of Spina Bifida, or GAPSBiF, an organization dedicated to increasing awareness and advocating for the prevention of spina bifida through large-scale food fortification with folic acid. It has affected change in fortification strategies around the world—Pakistan and Ethiopia earlier this year became the latest countries to commit to fortification. The Corn Masa Fortification Bill represents success closer to home. “It basically patches a hole in national fortification strategies and will protect children in cultures where corn is the predominant carbohydrate staple in the diet,” Blount said. The cause of spina bifida is not fully understood, but researchers know that nutritional shortage in folic acid is the most important contributor to spina bifida prevalence worldwide. “I am utterly thrilled about these advances, as they mean that children will not get these birth defects,” Blount said. Coyne-Beasley Elected APS President Elect. Tamera Coyne-Beasley, M.D., MPH, professor in the Division of Adolescent Medicine, was elected as the 2025–2026 president-elect of the American Pediatric Society (APS) and began serving her term in June. Founded in 1887, APS aims to advance child and adolescent health and well being through an engaged, diverse and inclusive community of pediatric thought LEADERSHIP

HONORS

Schmitt Gives Prestigious Lecture. At the Pediatric Endocrinology Society (PES) Annual meeting in May,

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