FDA Approves Fayuvi Gene Therapy for Rare Sanfilippo SyndromeNovember 1, 2026 Health 4 Views The FDA has approved Fayuvi, a gene therapy for Sanfilippo syndrome, a rare inherited disease. The treatment costs nearly $4 million per patient.
FDA Approves First Drug for Rare Alexander DiseaseOctober 13, 2026 Health 15 Views The FDA has approved Zanvastro, the first disease-modifying drug for Alexander disease, a rare neurological disorder. The treatment targets the underlying genetic cause to stabilize motor function.
