CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and…
Articles by Joana Carvalho, PhD
PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising…
Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of…
The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the…
Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test…
Promising safety and tolerability data from a Phase 1 trial on suvodirsen (WVE-210201), Wave Life Sciences‘ investigational exon-skipping therapy…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for …
Sarepta Therapeutics‘ casimersen (SRP-4045), one of the company’s investigational exon-skipping therapies for Duchenne muscular dystrophy (DMD), showed promising…
Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone —…
Catabasis Pharmaceuticals will collaborate with the University of Texas Southwestern to investigate potential benefits of edasalonexent on heart…