FDA Clears Regeneron Bone Disease Treatment and Ultragenyx Gene Therapy

By Ed Silverman · STAT News · 2026-08-20

STAT News reports that federal regulators have authorized two novel treatments for ultra-rare conditions. Regeneron Pharmaceuticals received a green light for Pasatru to treat a bone growth disorder, while Ultragenyx secured approval for Genglycos, the first gene therapy targetin

STAT News reports that federal regulators have authorized two novel treatments for ultra-rare conditions. Regeneron Pharmaceuticals received a green light for Pasatru to treat a bone growth disorder, while Ultragenyx secured approval for Genglycos, the first gene therapy targetin