Clinical Trials

Blog Post

Clinical Research Alert: Phase 1 Study of Gene Therapy in Individuals with Myotonic Dystrophy Type 1

By MDA Staff | December 19, 2025
Blog Post

Clinical Research Alert: Two Phase 4 Studies of Risdiplam in Pediatric SMA Patients Following Gene Therapy

By MDA Staff | December 17, 2025
Blog Post

Clinical Research Alert: Phase 1/2 Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1

By MDA Staff | December 17, 2025
Clinical Research Phase 1b Study
Blog Post

Clinical Research Alert: Phase 1b Study of SRD-001 in Adults with DMD-Related Cardiomyopathy

By MDA Staff | December 5, 2025
A 3d rendering of a mitochondrion, the powerhouse of the cell, intertwined with a segment of DNA.
Blog Post

Simply Stated: Updates in Primary Mitochondrial Diseases

By Sujatha Gurunathan | November 26, 2025
Blog Post

Clinical Research Alert: Natural History Study of Individuals with Cardiomyopathy Associated with FRDA

By MDA Staff | November 21, 2025
Blog Post

Clinical Research Alert: Real World Study of Male Fertility Following Risdiplam Treatment

By MDA Staff | November 19, 2025
Illustration of a human brain, including the bulb-like structure in the lower part of the brain
Featured Article

Understanding Spinal-Bulbar Muscular Atrophy

By Matt Schur | November 14, 2025
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