Drug Development

Illustration of a single-stranded RNA curling out of a tangle of ribbon-like strands.
Featured Article

RNA Therapies Offer Keys to Treating Genetic Neuromuscular Diseases

By Andrew Zaleski | February 23, 2026
A compass, with the needle pointing north, rests on a rocky shore.
Featured Article

The New Diagnostic Journey: Advances in Genetic Testing Speed Rare Disease Diagnosis

By Amy Bernstein | February 23, 2026
Research Lab Scientist Using Computer. Medical Science
Blog Post

Everything You Need to Know About Clinical Trials for Neuromuscular Diseases

By Amy Bernstein | December 23, 2025
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Blog Post

In Case You Missed It…

By MDA Staff | December 8, 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
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