rare diseases
The latest news about rare diseases: headlines, AI summaries and the full story from every source.
- Disc Medicine shares rise ahead of Phase 3 APOLLO trial results
- FDA Launches Rare Disease Framework Initiative
- Mirum Pharmaceuticals Announces Topline Results for Livmarli Trial
- BridgeBio Reports Positive Phase 3 Trial Results for Rare-Disease Drug Encaleret
- Rocket Pharmaceuticals Advances Phase 2 Trial for RP-A501 in Danon Disease
- CSL partners with Alentis for lixudebart in rare kidney and liver diseases
- NORD Awards Seed Grants to Support Research on Four Rare Diseases
- FDA Approves Mirum and Incyte's Atebrioz for Rare Bone Disorder FOP
- FDA Approves Fayuvi for Sanfilippo Syndrome, Impact on Ultragenyx's Profitability Target
- FDA and Duke-Margolis Host Workshop on Evidence Standards for Rare Diseases
- BioCryst (BCRX) Posts First Profit and Eyes Expansion Through Rare Disease Acquisitions
- Intellia Therapeutics Receives FDA Priority Review for Lonvoz
- FDA Approves Regeneron's Rare-Disease Drug Pasatru Despite Small Market Potential
- Takeda receives FDA approval for rusfertide as treatment for polycythemia vera
- BioCryst Becomes Profitable and Seeks to Expand in Rare Disease Drug Market
- Jaguar Health Targets Mid-2027 NDA Filing for MVID with Potential End-2027 U.S. Approval
- BioMarin Acquires Alesta Therapeutics for $275M to Expand Rare Disease Pipeline
- New Medicaid Work Requirements Pose Risk to Rare Disease Patients
- Biogen Exceeds Quarterly Estimates with Strong Performance from Newer Drugs
- BioMarin and n-Lorem Foundation Collaborate on Treatment for Rare Genetic Disorder ReNU Syndrome
- Warburg Pincus-Led Investor Group Acquires Controlling Stake in PANTHERx Rare
- Vertex Pharmaceuticals to Acquire Crinetics Pharmaceuticals for $10 Billion
- Amgen Faces Potential EU Withdrawal of Tavneos Due to Data Integrity Issues
- Chan Zuckerberg Initiative's Biohub Announces Fourth Round of Rare As One Funding
- Sanofi Halts Phase 3 Trial of Riliprubart in Rare Nerve Disease Due to Efficacy Concerns
- Citizens JMP Securities Upgrades Rezolute Following Late-Stage Trial Update
- Savara Inc. Reports Q1 Financial Results
- Mirum Pharmaceuticals Reports Strong Revenue Growth and Raised Guidance for 2026
- BioMarin's Phase 3 Trial of BMN 401 Meets Only One Primary Endpoint
- Rocket Pharmaceuticals Reports Q1 2026 Net Loss of $47.6M
- Rare Disease Families Gain Tools for Drug Development Through Ultragenyx Bootcamps
- SuperTruth Launches Sean's Friends Donor Advised Fund, Pledges to Redirect Pediatric Revenue to Fund
- KalVista Pharmaceuticals Surges on Acquisition Announcement by Chiesi Group
- Polaryx Therapeutics to Participate in NTSAD Annual Family Conference
- Travere Therapeutics Shares Rise 40.8% Following FDA Approval of Filspari
- FDA Accepts BLA for Ultragenyx's Gene Therapy for Sanfilippo Syndrome