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Genetic Disorders

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

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

The findings point to gaps in specialist access on Long Island.

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