Clinical Trials

Blog Post

Clinical Research Alert: Phase 3 Study of RGX-202 in Boys with DMD

By MDA Staff | June 30, 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 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
ALS Amyotrophic Lateral Sclerosis - progressive nervous system disease. Amyotrophic lateral sclerosis. Day background design.
Blog Post

Simply Stated: Updates in Amyotrophic Lateral Sclerosis (ALS)

By Sujatha Gurunathan | April 29, 2025
Blog Post

Clinical Research Opportunity: LION-CS101 a Phase 1/2 Study of AB-1003 in Adults with LGMD2I/R9

By MDA Staff | March 31, 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
Medical illustration of musculature in the upper back, shoulders, neck, and head.
Featured Article

Stem Cell Innovations Drive Advances in Muscle Regeneration

By Andrew Zaleski | February 28, 2025
Composite image of scientific lab instruments: a microscope, a pipette, petri dishes, and a computer keyboard.
Featured Article

Innovations in GNE Myopathy Research

By Amy Bernstein | February 28, 2025
Blog Post

Clinical Trial Alert: Phase 2/3 Study of an Investigational Medication in Pediatric Patients with Generalized Myasthenia Gravis

By MDA Staff | February 19, 2025