INmune Bio's high-upside potential with CORDStrom for RDEB and XPro for Alzheimer's, offering up to 144.8x returns. Click ...

Bexobrutideg, a first-in-class Bruton’s tyrosine kinase degrader, has been granted orphan drug designation from the FDA in ...

Alexion Pharmaceuticals’ ravulizumab for the prevention of delayed graft function after renal transplantation was granted FDA orphan ...

UK-based AstraZeneca’s acquired rare disease drug has met the primary endpoint in a Phase III trial in patients with chronic ...

Health Canada has granted approval for Biogen’s Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA), a rare, ...

Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030. The 2025 ...

Completed dosing of first two cohorts in Phase 1/2a REWRITE Clinical Study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD); Interim ...

Batoclimab is an anti-FcRn treatment being developed for a range of autoimmune diseases and active thyroid eye disease (TED).