Clinical In Vivo Gene Therapy Patients Stories Listing

Our experts are at the forefront of clinical in vivo gene therapy breakthroughs.

Patient story

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

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.

Patient story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.

Patient story

Experimental Gene Therapy for Hemophilia B: Jay’s Story

For 50-plus years, Jay dealt with the frequent treatments needed for his hemophilia. A gene therapy put an end to the treatments and to the worry the disease caused him.

Patient story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

For nearly 50 years, Bill needed frequent treatment for hemophilia — often every few days. A gene therapy changed everything.

Patient story

William’s Story: Gene Replacement Therapy for SMA Type 2

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.

Patient story

Gene Therapy Treatment for Spinal Muscular Atrophy: Céline’s Story

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

Patient story

Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story

Diagnosed with Leber congenital amaurosis as a baby, Hannah was on a path to going blind until gene therapy at Children’s Hospital restored her sight.

Patient story

Spinal Muscular Atrophy Treatment: Claire’s Story

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

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Clinical In Vivo Gene Therapy Patients Stories Listing

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Experimental Gene Therapy for Hemophilia B: Jay’s Story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

William’s Story: Gene Replacement Therapy for SMA Type 2

Gene Therapy Treatment for Spinal Muscular Atrophy: Céline’s Story

Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story

Spinal Muscular Atrophy Treatment: Claire’s Story