Research

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

Quest Podcast: Voices of Inclusion: Celebrating NDEAM with Disability:IN

By MDA Staff | October 21, 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
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Blog Post

In Case You Missed It…

By MDA Staff | September 8, 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
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Blog Post

In Case You Missed It…

By MDA Staff | July 8, 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