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Rare Diseases

Epidermolysis Bullosa Menkes Disease Hunter Syndrome Barth Syndrome Activated PI3-Kinase Delta Syndrome (APDS)

FDA Approves Zycubo, First U.S. Treatment for Menkes Disease

Early use cut mortality by nearly 80%, offering a targeted option for a fatal copper-transport disorder.

FDA Grants Accelerated Approval to Forzinity, First Therapy for Barth Syndrome