Larimar’s Nomlabofusp Shows Long-Term Promise in Friedreich’s Ataxia
Bala Cynwyd, Pa., September 29, 2025: Larimar Therapeutics announced positive new data from an ongoing long-term open-label study of...
rare disease
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FDA Accelerates First-Ever Barth Syndrome Therapy, FORZINITY™
NEEDHAM, Mass., September 19, 2025 – Stealth BioTherapeutics Inc. today announced that the U.S. Food and Drug Administration (FDA)...
FDA Grants Fast Track Status to NS-229 for Rare Autoimmune Disease
PARAMUS, NJ, September 9, 2025 — The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to...
UK Approves Once-Monthly Octreotide Injection for Acromegaly Care
Lund, Sweden — August 28, 2025 — The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval...
FDA Approves First RNA Therapy for Hereditary Angioedema Prophylaxis
CARLSBAD, Calif., August 21, 2025 — The U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen), the first...
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