Tag
Rare diseases
- Ultragenyx Pharmaceutical agrees to US$203M priority review voucher sale
- Pharming Group reports 26.4% spleen reduction in Phase 2 leniolisib trial
- Mirum Pharmaceuticals reports LIVMARLI met Phase 3 EXPAND endpoint
- BridgeBio Pharma reports symptom gains in Phase 3 encaleret trial
- Ultragenyx says EMA validates Sanfilippo gene therapy application
- Ultragenyx plunges after-hours from rare disease drug trial failure
- Neurogene patient dies in drug trial, stock surges after FDA says 'use lower dose'
- Capricor Therapeutics gains traction with impending treatment data reveal in Czechia
- Ontario scientists create AI algorithm to identify rare disorders in kids