Browsing Tag
rare disease
23 posts
Rhythm Pharmaceuticals (RYTM) surges 35% after announcing Phase 2 trial data reveal for oral MC4R agonist bivamelagon
Find out why Rhythm Pharmaceuticals stock surged over 35% and what the upcoming bivamelagon trial results mean for its rare disease pipeline.
CSL wins FDA approval for ANDEMBRY, the first monthly anti-factor XIIa therapy for hereditary angioedema
CSL’s ANDEMBRY gains U.S. FDA approval as the first monthly HAE treatment targeting factor XIIa. Learn how it redefines disease control for patients.
FDA committee endorses Zevra Therapeutics’ arimoclomol as a treatment for Niemann-Pick Disease Type C
In a notable advancement for rare disease treatment, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) has received a significant endorsement…