Gene Therapy

Blog Post

Simply Stated: What is Spinal Muscular Atrophy?

By Jeanene Swanson | August 15, 2019
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From Drug Discovery to Delivery

By MDA Staff | July 15, 2019
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Guiding Treatment for Myotonic Dystrophy

By MDA Staff | July 15, 2019
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Ongoing Clinical Trials

By Ori Rokach | July 15, 2019
Featured Article

The State of Gene Targeted Therapies

By Jeanene Swanson | July 15, 2019
Picture of two hands using a screwdriver to disassemble a strand of DNA
Featured Article

These 7 Things Can Help You Understand Gene Targeting Therapy

By Amy Madsen | July 15, 2019
Blog Post

MDA Awards Venture Philanthropy Funding to Locana to Develop Novel Treatment for DM

By Jeanene Swanson | June 12, 2019
Illustration of DNA
Blog Post

FDA Approves AveXis’ Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients

By Jeanene Swanson | May 24, 2019
Blog Post

Do You Know ALS? Meet Dr. Glass.

By MDA Staff | May 22, 2019
Blog Post

Do You Know ALS? Read About the Latest Research.

By Jeanene Swanson | May 15, 2019