Gene Therapy

Blog Post

Five Questions with LGMD Researcher Martin Childers

By Jeanene Swanson | September 30, 2019
baby girl
Blog Post

Biogen Announces Positive Long-Term Results from Phase 2 Study of Spinraza to Treat SMA in Pre-Symptomatic Infants

By Jeanene Swanson | August 21, 2019
Blog Post

Simply Stated: What is Spinal Muscular Atrophy?

By Jeanene Swanson | August 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
Featured Article

From Drug Discovery to Delivery

By MDA Staff | July 15, 2019
Featured Article

Guiding Treatment for Myotonic Dystrophy

By MDA Staff | July 15, 2019
Featured Article

Ongoing Clinical Trials

By Ori Rokach | 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