FDA grants Breakthrough Therapy Designation to Hemab Therapeutics’ sutacimig for Glanzmann thrombasthenia. Discover what this means for bleeding disorder treatment.
Early YOLT-202 data from YolTech Therapeutics suggests gene editing could redefine Alpha-1 Antitrypsin Deficiency treatment. Read the analysis.
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
Waskyra’s FDA and EMA approvals spotlight AGC Biologics’ rare disease manufacturing model. Find out what this means for future nonprofit-led therapies.
Vonafexor reversed kidney decline in Alport syndrome patients. Find out what ENYO Pharma’s Phase 2 results could mean for FXR drugs and kidney care.
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