Clinical Trials

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
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