Drug Approval

Adrian Krainer, PhD
Blog Post

Letter from the Editor: How New Higher-Dose Regimen for Spinraza Brings More Hope

By Mindy Henderson | March 30, 2026
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Featured Article

Expert Tips for Handling an Insurance Claim Denial for Gene Therapy

By Emily Blume | February 23, 2026
Illustration of a cross-section of a mitochondria, a sausage-shaped organelle that contains strands of mitochondrial DNA.
Featured Article

Understanding Thymidine Kinase 2 Deficiency

By Michelle Jackson | February 23, 2026
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Blog Post

In Case You Missed It…

By MDA Staff | December 8, 2025
Blog Post

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

By MDA Staff | September 24, 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
Blog Post

Quest Podcast: PJ’s Protocol: A Lifesaving Procedure Fueled by Love

By MDA Staff | March 6, 2025
Medical Research Scientist Working on Desktop Computer with Gene Analysis Software in Modern Science Research Laboratory. Lab Engineers in White Coats Conduct Experiments in the Background.
Blog Post

Simply Stated: Emerging Therapies to Treat DMD

By Sujatha Gurunathan | December 20, 2024
Blog Post

MDA Ambassador Guest Blog: Finding Hope and Optimism in New Treatments

By Chase Rankin | June 4, 2024