Tag: #raredisease

Ultragenyx Agrees to Sell FDA Rare Pediatric-Disease Priority Review Voucher for $203 Million

Ultragenyx said it agreed to sell a rare pediatric disease priority review voucher tied to gene therapy FAYUVI for $203M, its second PRV sale this month.

$RARE, #biotech, #ultragenyx, #prv, #raredisease

Garetosmab (Pasatru) sharply reduces new bone lesions in adults with FOP, phase 3 Lancet trial shows; FDA approved Aug. 19, 2026

Phase 3 Lancet trial shows garetosmab cuts new heterotopic bone lesions by ~90% in adults with FOP; FDA approved Pasatru Aug. 19, 2026.

#fop, #garetosmab, #raredisease, #fda

Open-source AI improved doctors' rare-disease diagnoses in trial — but increased high-confidence errors

Preprint: open-source model RaDaR raised physicians’ rare-disease diagnostic accuracy by 21.4 points in a randomized case-based trial — but produced more high-confidence errors.

#ai, #raredisease, #diagnosis, #medicalai

Neurocrine to Buy Soleno for $2.9 Billion, Picks Up Prader‑Willi Drug VYKAT XR

Neurocrine will acquire Soleno for $2.9B to add VYKAT XR, the newly approved Prader‑Willi hyperphagia drug, aiming to expand its rare‑disease portfolio.

#biotech, #m&a, #raredisease