Neuromuscular Research, Central Florida
- About Us
- Our Research
Advancing Care & Discovery for Children With Neuromuscular Disorders
The Neuromuscular Research team at Nemours Children’s in Central Florida brings together experts in neurology, rehabilitation, and respiratory care with one shared goal: to help children with nerve and muscle disorders move, breathe, and live more independently.
We lead and participate in clinical and observational trials that expand access to breakthrough treatments for Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), Charcot-Marie-Tooth (CMT) disease, and related neuromuscular conditions.
Through close collaboration with national research networks, academic partners, and industry sponsors, we move discoveries from the lab to the clinic faster, giving families earlier access to promising new therapies.
Pioneering Pediatric Neuromuscular Research
We’re advancing innovation in pediatric neuromuscular care through gene-based therapies, clinical trials, and collaboration. Our numbers tell the story.
8+
Active clinical and observational trials
2
National Centers of Excellence designations
2
Gene therapy platforms under study
We partner across our system and national networks to share genetic data, biomarkers, and clinical insights.
As leaders in research-driven care, we’re certified by the Muscular Dystrophy Association (MDA) and recognized as a Center of Excellence by the Charcot-Marie-Tooth Association (CMTA) and the Myasthenia Gravis Foundation.
Our investigators have played key roles in clinical trials supporting Food and Drug Administration (FDA)-approved therapies that shape the future of pediatric neuromuscular medicine.
We integrate rehabilitation therapy, respiratory care, and family-centered approaches into research to improve quality of life and long-term independence.
Our Leadership
Omer Abdul Hamid, MD
Director, Neuromuscular Research, Central Florida
Research Team
Not listed below: Wendy Leon, BSN, RN, CCRC, Clinical Research Nursing Lead
Innovation in Pediatric Neuromuscular Research
Our program focuses on understanding the genetic and molecular basis of neuromuscular diseases. Every project connects research with real-world clinical care to improve how we treat and support children.
Focus Areas
We focus on these key areas of research:
- Gene-based therapies: Developing and refining treatments that address the genetic causes of neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy.
- Clinical and observational trials: Leading and participating in studies that test new therapies, medications, and devices for children with neuromuscular conditions.
- Early detection and disease tracking: Studying how symptoms develop and progress to support earlier diagnosis and more precise, personalized care.
- Rehabilitation and respiratory outcomes: Integrating physical therapy and breathing support into research to help children improve mobility and independence.
- Data and registry collaboration: Sharing genetic and clinical data through national registries to improve care standards.
Research Themes
Our work is guided by core themes that shape every study:
- Translating genetic discoveries into practical treatments for kids.
- Personalizing care plans to match each child’s needs and goals.
- Integrating disciplines, including neurology, rehabilitation, and genetics, for a whole-child approach.
- Partnering to expand access and accelerate research progress.
Clinical Trials & Research Studies at Nemours
Pediatric clinical trials and research studies are important for advancing medical knowledge and improving care for kids around the world. Our researchers lead clinical research to study new drugs, treatments, devices, and techniques. We also contribute to national registries and collaboratives to further research in this area. Find a clinical trial or study now.
Awards & Recognition
Our program has earned national recognition for excellence in research, clinical care, and collaboration. These honors reflect our team’s commitment to advancing science and improving outcomes for children with neuromuscular disease.
Centers of Excellence Designations & Certifications
In Central Florida, Nemours is nationally recognized for excellence in pediatric neuromuscular care and research through the following designations:
- Charcot-Marie-Tooth Association (CMTA) Center of Excellence: Recognized for providing exceptional care and advancing research for children with Charcot-Marie-Tooth disease.
- Muscular Dystrophy Association (MDA)-Certified Program: Certified by the MDA for leadership in pediatric neuromuscular care and clinical research.
- Myasthenia Gravis Center of Excellence: Designated for expertise in diagnosing, managing, and researching childhood myasthenia gravis.
Collaborative Leadership in National Trials
Our investigators have contributed to landmark studies supporting FDA-approved therapies including Evrysdi®, Spinraza®, Zolgensma®, Agamree®, and Duvyzat®.
Related Research & Clinical Focus
Research at Nemours often aligns with multiple research areas, affiliated labs, and clinical programs to support discovery and care systemwide.
Genetics
CLINICAL CARE
Expert evaluation, diagnosis, and counseling for children with inherited or rare conditions, giving families clear answers and coordinated care.
Neuromuscular Program
CLINICAL CARE
Certified by the Muscular Dystrophy Association (MDA), Nemours specialists help kids with neuromuscular disorders live their best lives.
Neuroscience Research
RESEARCH AREA
We investigate brain development and neurological disorders to advance pediatric neuroscience through collaboration and translational science.
Occupational Therapy
CLINICAL CARE
Specialized pediatric care that develops the skills children need for daily life, promoting independence at home, in school, and in the community
Physical Therapy
CLINICAL CARE
Expert care that restores movement and builds strength after injury, surgery, or developmental delay, helping children reach their fullest physical potential.
Pulmonology
CLINICAL CARE
Comprehensive care for children with lung, airway, and breathing disorders provided by Nemours pediatric pulmonologists.