Rare Disease stories

Mar 26, 2026

FDA Approves Rocket Pharmaceuticals Gene Therapy for Rare Immune Disorder

The FDA approved Kresladi, Rocket Pharmaceuticals' gene therapy for a rare immune disorder, despite testing in only nine patients. The approval represents a notable decision as the agency has recently taken tougher stances on rare disease drugs.

Mar 26, 20263 sources

Mar 25, 2026

FDA Approves Denali Therapeutics Drug for Hunter Syndrome

The FDA approved a new medicine from Denali Therapeutics for Hunter syndrome, a rare genetic disorder. The approval is notable following a recent string of FDA rejections for rare disease drugs.

Mar 25, 20261 source