Neuromuscular Program Patient Stories

Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.

Cooper was one of the first at CHOP to receive a new FDA-approved infusion therapy for Duchenne muscular dystrophy, which is given to him at home. Results are promising.

Untreated, children with SMA type 2 will never be able to walk. After receiving a one-time dose of gene therapy, William has gained strength and hit milestones his family previously didn't think possible. 

Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy (SMA) at CHOP.

Diagnosed with myasthenia gravis, which causes eye drooping and double vision, Michaela is grateful for the team at CHOP for helping her see the world with both eyes wide open.

When medicine alone couldn’t successfully control Tajh’s myasthenia gravis symptoms, surgery to remove his thymus offered the best chance of remission.

With a breakthrough drug for spinal muscular atrophy treatment and ongoing therapy at Children’s Hospital of Philadelphia, Claire is reaching new milestones.

For the first 10 years of his life, Lucas had limited use of his right hand due to cerebral palsy. A surgery at CHOP gave him the mobility and flexibility that Lucas had always hoped for.