91大神

The Neuromuscular Research team at 91大神 Children鈥檚 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.</p>\r\n<p>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.</p>\r\n<p>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.</p>\r\n"}} id=text-70e7a3c9f9 class=cmp-text>

The Neuromuscular Research team at 91大神 Children鈥檚 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.

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.</p>\r\n<h2>Focus Areas</h2>\r\n<p>We focus on these key areas of research:</p>\r\n<ul>\r\n<li><b>Gene-based therapies:</b> Developing and refining treatments that address the genetic causes of neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy.</li>\r\n<li><b>Clinical and observational trials:</b> Leading and participating in studies that test new therapies, medications, and devices for children with neuromuscular conditions.</li>\r\n<li><b>Early detection and disease tracking:</b> Studying how symptoms develop and progress to support earlier diagnosis and more precise, personalized care.</li>\r\n<li><b>Rehabilitation and respiratory outcomes:</b> Integrating physical therapy and breathing support into research to help children improve mobility and independence.</li>\r\n<li><b>Data and registry collaboration:</b> Sharing genetic and clinical data through national registries to improve care standards.</li>\r\n</ul>\r\n"}} id=text-a23a66e04f class=cmp-text>

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.

Our work is guided by core themes that shape every study:</p>\r\n<ul>\r\n<li>Translating genetic discoveries into practical treatments for kids.</li>\r\n<li>Personalizing care plans to match each child鈥檚 needs and goals.</li>\r\n<li>Integrating disciplines, including neurology, rehabilitation, and genetics, for a whole-child approach.</li>\r\n<li>Partnering to expand access and accelerate research progress.</li>\r\n</ul>\r\n"}} id=text-665189fe05 class=cmp-text>

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鈥檚 needs and goals.
• Integrating disciplines, including neurology, rehabilitation, and genetics, for a whole-child approach.
• Partnering to expand access and accelerate research progress.

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.</p>\r\n"}} id=text-6228c9cb7d class=cmp-text>

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.

Our program has earned national recognition for excellence in research, clinical care, and collaboration. These honors reflect our team鈥檚 commitment to advancing science and improving outcomes for children with neuromuscular disease.</p>\r\n<h3><b>Centers of Excellence Designations &amp; Certifications</b></h3>\r\n<p>In Central Florida, 91大神 is nationally recognized for excellence in pediatric neuromuscular care and research through the following designations:</p>\r\n<ul>\r\n<li><b>Charcot-Marie-Tooth Association (CMTA) Center of Excellence:</b> Recognized for providing exceptional care and advancing research for children with Charcot-Marie-Tooth disease.</li>\r\n<li><b>Muscular Dystrophy Association (MDA)-Certified Program:</b> Certified by the MDA for leadership in pediatric neuromuscular care and clinical research.</li>\r\n<li><b>Myasthenia Gravis Center of Excellence:</b> Designated for expertise in diagnosing, managing, and researching childhood myasthenia gravis.</li>\r\n</ul>\r\n<h3><b>Collaborative Leadership in National Trials&nbsp;</b></h3>\r\n<p>Our investigators have contributed to landmark studies supporting FDA-approved therapies including Evrysdi庐, Spinraza庐, Zolgensma庐, Agamree庐, and Duvyzat庐.</p>\r\n"}} id=text-022fc3fbb1 class=cmp-text>

Our program has earned national recognition for excellence in research, clinical care, and collaboration. These honors reflect our team鈥檚 commitment to advancing science and improving outcomes for children with neuromuscular disease.

Centers of Excellence Designations & Certifications

In Central Florida, 91大神 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听

Our investigators have contributed to landmark studies supporting FDA-approved therapies including Evrysdi庐, Spinraza庐, Zolgensma庐, Agamree庐, and Duvyzat庐.

Research at 91大神 often aligns with multiple research areas, affiliated labs, and clinical programs to support discovery and care systemwide.</p>\r\n"}} id=text-94ffcf693b class=cmp-text>

Research at 91大神 often aligns with multiple research areas, affiliated labs, and clinical programs to support discovery and care systemwide.