Joana Carvalho, PhD, managing science editor —

CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD), significantly improved skeletal muscle and lung function in a group of boys and young men at advanced stages of the disease, top-line data from an interim analysis of a Phase 2 trial show. CAP-1002…

PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising safety and efficacy results in a small group of boys with DMD, a Phase 1b trial shows. The company presented the preliminary findings at the 25th Annual Parent Project…

Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people with LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). LAMA2 MD, also known as merosin-deficient congenital…

The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells in patients with Duchenne and Becker muscular dystrophy. “Understanding the immune profile and how it changes through the course of disease can provide…

Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test that looks for specific markers in cells from a mother’s placenta, according to a small study of pregnant women whose children are at risk for this disease. A misdiagnosis, however,…

Promising safety and tolerability data from a Phase 1 trial on suvodirsen (WVE-210201), Wave Life Sciences‘ investigational exon-skipping therapy for boys with Duchenne muscular dystrophy (DMD), opens the door for the launch of new Phase 2/3 clinical trials later this year. Final results from the trial (NCT03508947) were…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for Duchenne muscular dystrophy (DMD) patients. Pamrevlumab is a human monoclonal antibody that blocks the activity of the connective tissue growth factor (CTGF), a protein involved in tissue scarring (fibrosis) linked…

Sarepta Therapeutics‘ casimersen (SRP-4045), one of the company’s investigational exon-skipping therapies for Duchenne muscular dystrophy (DMD), showed promising results in an interim analysis of an ongoing Phase 3 clinical trial. These positive data are expected to support the submission of a new drug application (NDA) with the…

Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone — on heart and muscle health, but with less detrimental side effects, a study in animal models of the disease reports. The study, “Vamorolone targets dual nuclear receptors to treat…

Catabasis Pharmaceuticals will collaborate with the University of Texas Southwestern to investigate potential benefits of edasalonexent on heart function in Duchenne and Becker muscular dystrophies. After the promising results of Catabasis’ Phase 1/2 MoveDMD trial (NCT02439216) and its open-label extension, edasalonexent is now being…