rare_diseases
The latest news about rare_diseases: headlines, AI summaries and the full story from every source.
- Arcturus Reports Positive Mid-Stage Trial Results for ARCT-810 in OTC Deficiency
- BioMarin Acquires Alesta Therapeutics for $275 Million to Expand Rare Disease Pipeline
- BioMarin Acquires Alesta Therapeutics for $275 Million in Deal with Potential Additional Payments
- Mirum Pharmaceuticals Reports Positive Phase 2b Data for Volixibat in PSC
- BioMarin's Phase 3 Trial of BMN 401 for ENPP1 Deficiency Meets One Co-Primary Endpoint
- Arcturus Reports Q1 2026 Earnings and Clinical Progress