Can volixibat and brelovitug expand Mirum Pharmaceuticals’ rare liver disease ambitions?
Mirum Pharmaceuticals’ EASL 2026 data deepen its rare liver disease strategy. Find out what changes for PSC, HDV and PFIC care.
How Veld Pharmaceuticals and Chiesi Farmaceutici are expanding rare disease therapy access in Africa
Chiesi Farmaceutici and Veld Pharmaceuticals expand rare disease therapy access across Africa. Discover what this means for market access and regulation.
Acadia Pharmaceuticals broadens Rett syndrome treatment flexibility with DAYBUE STIX rollout in the United States
Acadia Pharmaceuticals has launched DAYBUE STIX for Rett syndrome in the U.S. Read what this means for adoption, tolerability, and market strategy.
Can Acadia overturn EMA’s negative opinion on trofinetide for Rett syndrome?
Acadia seeks EU re-examination after CHMP rejects trofinetide for Rett syndrome. Read the regulatory and clinical implications.
What Orphalan’s Orphelia Pharma acquisition reveals about the next phase of rare pediatric drug consolidation
Orphalan, the international pharmaceutical company known for developing orphan drugs, has announced the acquisition of Orphelia Pharma, a specialist in rare and severe pediatric diseases. The deal enhances Orphalan’s position in the European rare disease market, particularly within pediatric neurology and oncology, and signals a strategic pivot toward deeper vertical integration in pediatric formulations and […]