Infrequent
Amylyx Reveals Interim Rare Disease Data for Withdrawn ALS Therapy
Amylyx, Rare disease data, ALS drug withdrawal, Interim results, Rare neurological disorder
Eiger BioPharmaceuticals Declares Bankruptcy and Announces Operational Wind Down Following Years of Challenges
Eiger BioPharmaceuticals, Bankruptcy, Chapter 11, Assets Sale, Operational Wind Down, Rare Metabolic Disease Therapy Development
AstraZeneca’s Ultomiris Gains FDA Approval for Rare Autoimmune Condition – NMOSD
AstraZeneca, Ultomiris, ravulizumab-cwvz, FDA Approval, Rare Autoimmune Disease, Neuromyelitis Optica Spectrum Disorder (NMOSD), Long-acting C5 complement inhibitor, Phase 3 CHAMPION-NMOSD Trial, Reduced Relapse Rate, Dosing Frequency
Takeda’s $5.2B Ariad Deal Yields Label Expansion for Iclusig in Rare Leukemia
Takeda, Ariad, $5.2B deal, Iclusig, Label expansion, Rare form of leukemia