Rare Diseases

Gene Therapy FDA Approval for Rare Disease: Updated List and Analysis

FDA · rare disease · 5 min

FDA Priority Review Voucher Program: Will Makary's Initiative Survive?

FDA · 7 min

BioMarin Rare Disease Therapy Fails Phase 3: Key Takeaways for Pharma Teams

FDA · rare disease · 6 min

FDA Hold Puts Aardvark Prader-Willi Drug in Limbo: Implications for Pharma BD

FDA · Prader-Willi syndrome · 8 min

Servier Acquires Edgewise Therapeutics' Muscular Dystrophy Assets in Potential $2.7 Billion Deal

muscular dystrophy · 13 min

Immunovant's IMVT-1402 Shows Blockbuster Potential in Autoimmune Disease; Wave Life Sciences Advances RNA Editing

FDA · autoimmune disease · 9 min

Global Regulatory Roundup: Key Updates for June 1, 2026

FDA · Dermatologicals · 7 min

Palvella Secures $230M to Advance QTORIN for Rare Diseases

rare disease · 2 min

Palvella's Pivotal Rare-Disease Data and Financial Outlook

rare disease · 4 min

Palvella's Nasdaq Uplisting: Implications for Rare Disease Development

rare disease · 3 min

Palvella's Pivotal Data and Cash Runway: Implications for Rare Diseases

rare disease · 5 min

Fierce Biotech Week: Key Takeaways and Emerging Trends

biotech · 12 min