Gene Therapy

Illustration of a microscope and surrounded by beakers and test tubes.
Featured Article

Progress Now: Immunotherapy Study Recruiting, Drug Cleared for Expanded Access, and More

By MDA Staff | November 14, 2025
Illustration of a DNA double helix on a dark blue background
Featured Article

Say Yes to Gene Therapy? Factors to Consider When Gene Therapy Is an Option

By Amy Bernstein | November 14, 2025
Blog Post

Quest Podcast: Precision Medicine: Mapping the Genetic Code for New Treatments

By MDA Staff | September 24, 2025
Featured Article

Progress Now: Research News and Updates Across MDA Diseases

By MDA Staff | August 19, 2025
Graphic of laboratory equipment to signify neuromuscular disease research
Featured Article

Neuromuscular Disease Research: Going Strong

By Andrew Zaleski | August 19, 2025
Disabled Father play with his little son on the playground
Blog Post

Your Guide to Genetic Testing for Neuromuscular Diseases

By Megan Kramer-Salvitti | June 20, 2025
3d illustration showing molecules passing an ion channel in cell membrane
Blog Post

Simply Stated: Updates in Ryanodine Receptor 1-Related Disorders (RYR-1-RD)

By Sujatha Gurunathan | May 29, 2025
Illustration of the neuromuscular junction, where nerves send signals to muscles.
Featured Article

Understanding Congenital Myasthenic Syndrome: Causes and Treatments

By Justine Savage | May 23, 2025
Illustration of a researcher in a white lab coat climbing a ladder shaped like a DNA helix.
Featured Article

Why MDA’s Kickstart Program Is a Game-changer for Gene Therapies

By Amy Bernstein | May 23, 2025
Inside a hospital room, two healthcare providers wearing scrubs, gowns, masks, and head covers stand beside a bed where a young boy is lying down.
Featured Article

Milestone Moment: A Look Back at the First Gene Therapy Trial for DMD

By MDA Staff | February 28, 2025