Clinical Trials

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

Clinical Research Opportunity: Phase 3 Study of Cladribine in People with Generalized Myasthenia Gravis (gMG)

By MDA Staff | November 12, 2025
Blog Post

Clinical Research Alert: Phase 1 Study of AMX0114 in Adults with ALS

By MDA Staff | November 11, 2025
Arturito Estopinan, a teen who received a new therapy for the rare TK2d disease, seated in a wheelchair between his parents, Olga and Art.
Blog Post

How Expanded Access and Compassionate Use Broaden Access to Investigational Therapies

By Amy Bernstein | November 4, 2025
Blog Post

Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD

By MDA Staff | September 5, 2025
Blog Post

Clinical Research Alert: Phase 1/2 Study of SGT-003 in Boys with DMD

By MDA Staff | September 2, 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
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