MarketPulse

neuromuscular disease

4 articles
GlobeNewswire Inc.GlobeNewswire Inc.··Muscular Dystrophy Association

FDA Expands VYVGART Approval to All Generalized Myasthenia Gravis Patients

FDA expands VYVGART approval to include seronegative myasthenia gravis patients, broadening treatment access and increasing commercial opportunity for Ultragenyx.
ARGXFDA approvalclinical trial
GlobeNewswire Inc.GlobeNewswire Inc.··Argenx Se

argenx Expands VYVGART Leadership with Positive Phase 3 Data Across MG and CIDP

argenx presents Phase 3 data supporting VYVGART expansion across ocular MG, seronegative MG, and treatment-naïve CIDP, with 87.5% early benefit rates.
ARGXFDA approvalPhase 3 trials
GlobeNewswire Inc.GlobeNewswire Inc.··Na

Metagenomi's Compact CRISPR Breakthrough Achieves 64% Protein Knockdown in Mice

Metagenomi's compact CRISPR nuclease achieves 64% protein knockdown in mice via single AAV injection, validating its approach for non-hepatic genome editing therapies.
MGXin vivo gene editingneuromuscular disease
GlobeNewswire Inc.GlobeNewswire Inc.··Na

Dyne Therapeutics Launches Phase 3 Trial for Z-Basivarsen in Myotonic Dystrophy

Dyne Therapeutics initiates Phase 3 HARMONIA trial testing z-basivarsen in approximately 150 DM1 patients, aiming to convert accelerated approval to traditional FDA clearance.
DYNPhase 3 clinical trialFDA approval