Browse All

Current Filters

CLEAR FILTER x

TITLE

Evaluating Functional Changes and Outcome Measure Responsiveness in Limb Girdle Muscular Dystrophy: A 12-month Analysis of the 10-meter Run Time, NSAD, and PUL

90-month Muscle Function and Biomarker Outcomes With Cipaglucosidase Alfa Plus Miglustat (Cipa+Mig) in Adults With Pompe Disease in ATB200-02, an Open-label Phase I/II Study

Post-hoc Analysis of Descartes-08, BCMA-directed mRNA CAR-T Therapy, in Biologic Non-responsive Versus Biologic Naive Generalized Myasthenia Gravis: Results from a Phase 2b Randomized Trial