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Genetic Disorders

Spinal Muscular Atrophy Duchenne Muscular Dystrophy Severe Combined Immunodeficiency Sickle Cell Disease Cystic Fibrosis Erythropoietic Protoporphyria Fibrosis Cystica Stormorken Syndrome Telomeropathies Sanfilippo Syndrome Hemophilia Thalassaemia Mitochondrial Disorders

FDA Staff Flags Major Flaws in Capricor’s Deramiocel Data

An independent advisory committee will decide on July 29 whether the trial evidence gives regulators enough reason to approve the cell therapy.

NYU Study Finds Highest Sickle Cell Death Risk and Costs on Long Island

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy