Gene Therapy

Parents hold their four-year-old daughter, who received Zolgensma for spinal muscular atrophy as an infant.
Blog Post

A Revolutionary Decade for Spinal Muscular Atrophy Therapies

By Chris Anselmo | August 1, 2024
Bafus family photo of mom and dad each holding one small boy
Blog Post

My Gene Therapy Journey: The Bafus Family

By Rebecca Hume | July 9, 2024
Communication icons indicating connecting by phone, email, or video chat are grouped together in bubbles.
Featured Article

How to Get One-on-One Support From MDA

By MDA Staff | May 9, 2024
A group of people from neuromuscular advocacy organizations gathered at the 2024 MDA Clinical & Scientific Conference.
Featured Article

Understanding Titinopathies

By Myrna Traylor | May 9, 2024
Blog Post

Episode 39- Behind the Scenes: A Look at the Science and Research for New Treatments

By MDA Staff | April 1, 2024
Blog Post

Clinical Study Alert: A Phase 1/2 Study of Safety and Efficacy of LX2006 Gene Therapy in Adults with Cardiomyopathy Associated with Friedreich Ataxia

By Sujatha Gurunathan | March 25, 2024
Blog Post

At MDA Conference Neuromuscular Disease Experts Discuss Readiness for Gene Therapies

By Larry Luxner | March 20, 2024
Illustration of a black metal pipeline on a yellow background
Featured Article

What Is the Drug Development Pipeline?

By Amy Bernstein | February 12, 2024
Blog Post

Announcing the 2024 MDA Clinical & Scientific Conference Agenda

By MDA Staff | January 11, 2024
Blog Post

9 Questions to Ask Before Joining a Clinical Trial

By Myrna Traylor | January 11, 2024