Tag Archives: The Stem Cell Blog

Gene Therapy MYO-101 Receives FDA’s Orphan Drug Status for LGMD2E Treatment – Muscular Dystrophy News

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2018-04-27 NEWS

Muscular Dystrophy News

Gene Therapy MYO-101 Receives FDA’s Orphan Drug Status for LGMD2E Treatment
Muscular Dystrophy News
The U.S. Food and Drug Administration (FDA) granted orphan drug status to MYO-101, a gene therapy being developed by Myonexus Therapeutics for the treatment of limb girdle muscular dystrophy type 2E (LGMD2E) . The FDA’s orphan drug designation is

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2018-04-27 NEWS

Quartz

After gene-editing, Crispr’s new frontier to detect diseases is closer to becoming a reality
Quartz
The world is one step closer to using Crispr, a powerful gene-editing tool, to solve one of the hardest parts of being a doctor: figuring out what is wrong with a patient. While there are a range of diagnostic tools to help doctors with that very first
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