Rare Diseases

Monopar Starts Rolling NDA for ALXN1840 Wilson

FDA · Rare Diseases · 3 min

FDA Accepts BridgeBio Encaleret NDA for ADH1

FDA · Rare Diseases · 3 min

FDA Accepts Dyne BLA for z-Rostudirsen in DMD

FDA · Rare Diseases · 3 min

FDA Approved Gene Therapy List for Rare Disease

FDA · rare disease · 5 min

FDA Priority Review Voucher CNPV: Survival Odds

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 $2.65B Muscular Dystrophy Asset 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