Ehlers-Danlos Syndrome Spinal Muscular Atrophy Metabolic Disorders Epidermolysis Bullosa Cardiac Amyloidosis Chromosomal Disorders 5-Alpha-Reduktase Deficiency Hypertrichosis Congenital Insensitivity to Pain Trisomy Disorders
FDA fast-tracked the six-month custom CRISPR therapy that corrected KJ’s CPS1 mutation, enabling normal protein metabolism with reduced drug support.