Drug Development

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
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Featured Article

Say Yes to Gene Therapy? Factors to Consider When Gene Therapy Is an Option

By Amy Bernstein | November 14, 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
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Blog Post

In Case You Missed It…

By MDA Staff | October 8, 2025
Blog Post

Quest Podcast: Precision Medicine: Mapping the Genetic Code for New Treatments

By MDA Staff | September 24, 2025
Featured Article

Progress Now: Research News and Updates Across MDA Diseases

By MDA Staff | August 19, 2025
Blog Post

Behind the Drug: Nusinersen (Spinraza) for SMA

By Maggie Callahan | August 6, 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
Illustration of the neuromuscular junction, where nerves send signals to muscles.
Featured Article

Understanding Congenital Myasthenic Syndrome: Causes and Treatments

By Justine Savage | May 23, 2025