Clinical Trials

Blog Post

Behind the Drug: Nusinersen (Spinraza) for SMA

By Maggie Callahan | August 6, 2025
Blog Post

Clinical Research Alert: At-Home Research Study in People with DM1

By MDA Staff | August 4, 2025
Woman in electric wheelchair waits for consultation with doctor in clinic waiting area. Nurse invites patient with disability on procedures. Medical staff and people in modern medical facility lobby.
Blog Post

Behind the Drug: Risdiplam (Evrysdi) for SMA

By Maggie Callahan | July 31, 2025
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