Gene Therapy

An illustration of a doctor’s arm and hand holding tweezers and manipulating DNA.
Featured Article

Gene Editing Holds Promise as Viable Therapy

By Larry Luxner | November 18, 2024
2023 Clinical and Scientific Conference
Blog Post

MDA Kickstart: A New Hope for Ultra-Rare Neuromuscular Disease Treatments

By Sharon Hesterlee, Ph.D. | September 9, 2024
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Blog Post

In Case You Missed It…

By MDA Staff | September 8, 2024
A father, mother, and three sons sit on an outdoor patio step
Blog Post

My Gene Therapy Journey: The Curran Family

By Rebecca Hume | September 5, 2024
Illustration of a man walking into a DNA helix-shaped hedge maze.
Featured Article

Are You Ready for Gene Therapy? Here’s What You Need to Know

By Larry Luxner | August 13, 2024
A mom and dad hold their daughter and smile at her
Blog Post

My Gene Therapy Journey: The Graves Family

By Rebecca Hume | August 7, 2024
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
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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