Ultragenyx Gene Therapy Shows Durable Gains in MPS IIIA
NOVATO, Calif., February 3, 2026 — Ultragenyx Pharmaceutical Inc. has reported positive long-term clinical data for UX111, an investigational...
FDA accelerated approval
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Stealth BioTherapeutics Expands Access with Mito Assist™ and AnovoRx Partnership
NEEDHAM, Mass., October 9, 2025 — Stealth BioTherapeutics Inc. has announced the launch of Mito Assist™, a comprehensive patient...
Imlifidase Achieves Phase 3 Success in Kidney Transplant Trial
LUND, Sweden, Sept. 24, 2025 — Hansa Biopharma has announced that imlifidase successfully met its primary endpoint in the...
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 Adds Modeyso (dordaviprone) to Oncology Guidelines for H3 K27M-Mutant Diffuse Glioma
September 9, 2025 – In a significant advancement for neuro-oncology, Modeyso (dordaviprone) has been included in the National Oncology...
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