FDA Conditionally Approves Micro-dystrophin: A Small Gene With Big Promise
FDA Conditionally Approves Micro-dystrophin: A Small Gene With Big Promise https://pediatricsnationwide.org/wp-content/uploads/2023/04/Spring-23-Cover-Final-crop-1024x632.jpg 1024 632 Abbie Miller https://pediatricsnationwide.org/wp-content/uploads/2023/05/051023BT016-Abbie-Crop.jpgUPDATE: On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy…
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