Clinical In Vivo Gene Therapy Patient Stories
1 - 8 of 8
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.
Experimental Gene Therapy for Hemophilia B: Bill’s Story
![Experimental Gene Therapy for Hemophilia B: Bill’s Story Experimental Gene Therapy for Hemophilia B: Bill’s Story](https://www.chop.edu/sites/default/files/styles/teaser/public/experimental-gene-therapy-hemophilia-b-bill-s-story-0-16x9.jpg?itok=0t4BGXij)
Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.
Experimental Gene Therapy for Hemophilia B: Jay’s Story
![Experimental Gene Therapy for Hemophilia B: Jay’s Story Experimental Gene Therapy for Hemophilia B: Jay’s Story](https://www.chop.edu/sites/default/files/styles/teaser/public/experimental-gene-therapy-hemophilia-b-jay-s-story-16x9.jpg?itok=CbLZB5Ts)
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.
Experimental Gene Therapy for Hemophilia B: Bill’s Story
![Experimental Gene Therapy for Hemophilia B: Bill’s Story Experimental Gene Therapy for Hemophilia B: Bill’s Story](https://www.chop.edu/sites/default/files/styles/teaser/public/experimental-gene-therapy-hemophilia-b-bill-s-story-16x9.jpg?itok=D7u-xT5L)
For nearly 50 years, Bill needed frequent treatment for hemophilia — often every few days. A gene therapy changed everything.
William’s Story: Gene Replacement Therapy for SMA Type 2
![William William](https://www.chop.edu/sites/default/files/styles/teaser/public/neurology-gene-replacement-patient-william-320x180.jpg?itok=uBBgpAEt)
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.
Gene Therapy Treatment for Spinal Muscular Atrophy: Céline’s Story
![Celine Celine](https://www.chop.edu/sites/default/files/styles/teaser/public/Celine-16x9_0.jpg?itok=OkbbmIBB)
Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy (SMA) at CHOP.
Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story
![Hannah sitting and smiling Hannah sitting and smiling](https://www.chop.edu/sites/default/files/styles/teaser/public/hannah-ophthamology-patient-smiling-sitting-on-bench-780x439.jpg?itok=TNQ7lvbK)
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.
Spinal Muscular Atrophy Treatment: Claire’s Story
![Claire with her mom smiling Claire with her mom smiling](https://www.chop.edu/sites/default/files/styles/teaser/public/claire-sma-patient-with-mom-smiling-780x439.jpg?itok=sErdgepr)
With a breakthrough drug for spinal muscular atrophy treatment and ongoing therapy at Children’s Hospital of Philadelphia, Claire is reaching new milestones.