Micro-Dystrophin

Scientists restore muscle strength in dogs using micro-dystrophin gene therapy

Sarepta Therapeutics

Webinar: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (October 2021)

Parent Project Muscular Dystrophy

Duchenne Muscular Dystrophy and Dystrophin

In the Pipeline: Gene Therapy - Sarepta Therapeutics

Parent Project Muscular Dystrophy

022 - Sarepta Declines on Good Micro-Dystrophin Data

Breaking Biotech

DMD and Gene Therapy What's On the Horizon

The France Foundation

A film about some of the challenges in gene therapy, and a promising new approach

Gene Therapy for DMD How it Works and the Ongoing Clinical Trials

MDA

PORTRAIT OF DUCHENNE - Gene replacement therapy

Gene editing for muscular dystrophies

The Physiological Society

Introduction to Gene Therapy [PPMD Gene Therapy Webinar Series]

Parent Project Muscular Dystrophy

Webinar: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (June 2021)

Parent Project Muscular Dystrophy

ELEVIDYS and Genetic Variants

Parent Project Muscular Dystrophy

What does microdystrophin mean?

What Does That Mean?

Genes, Exons, and the Pathophysiology of DMD (Duchenne Muscular Dystrophy)

The France Foundation

Gene Therapy: The Power of Advocacy & Real World Evidence -- PPMD 2023 Annual Conference

Parent Project Muscular Dystrophy

FDA Approved Gene Therapy | Duchenne Muscular Dystrophy

NationwideChildrens

Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

The Children's Hospital of Philadelphia

An Analogy to Microdystrophin Gene Therapy

Timothy Kwan Ly

Duchenne Muscular Dystrophy (DMD) Mechanism of Disease

Sarepta Therapeutics