New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease

Retrieved on: 2024-11-19 20:50:37

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New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease. View article details on hiswai:

Excerpt

NCATS and NHGRI are teaming up to advance a gene therapy treatment for methylmalonic acidemia (MMA), a rare genetic disorder.

Article found on: ncats.nih.gov