rare disease
The latest news about rare disease: headlines, AI summaries and the full story from every source.
- FDA Approves Pimjetmi for Tenosynovial Giant Cell Tumor
- PepGen Shares Surge Following Positive Mid-Stage Trial Data for Myotonic Dystrophy Therapy
- ANI Pharma Reports Strong Q3 2026 Results Driven by Rare Disease Segment Growth
- ANI Pharmaceuticals Announces $560 Million Acquisition of Egetis Therapeutics
- AstraZeneca Reports Q3 2026 Results with Strong Oncology and Rare Disease Growth
- Inovio Pharmaceuticals Shares Surge Following FDA Approval of Voceryq for RRP
- Ultragenyx Receives Conditional UK Marketing Authorization for Dojolvi
- FDA Issues Draft Guidance to Accelerate Personalized Gene Therapy Development
- Immix Biopharma Reports Mid-Stage Trial Data for AL Amyloidosis Therapy