News

CureDuchenne, the Duchenne muscular dystrophy nonprofit  group engaged in disease advocacy and research funding,  announced on Sept. 7 — World Duchenne Awareness Day 2016 — that it has received a coveted STRIVE Award in recognition of its efforts. The awards were created by PTC Therapeutics to recognize, support, and honor…

A form of vitamin B3 taken as a supplement may reverse muscle frailty associated with aging by boosting levels of NAD, a molecule that helps generate energy from food nutrients, researchers report. The findings may lead to new ways of ameliorating certain aspects of muscle dystrophy. The recent study, “Loss of NAD…

Results from a Phase 3 clinical trial comparing deflazacort and prednisone, two corticosteroid drugs under evaluation as potential treatments for Duchenne muscular dystrophy, showed that both deflazacort and prednisone improved muscle strength compared to a placebo, meeting the primary endpoint of the study. But deflazacort caused less weight gain than prednisone, according to…

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Mallinckrodt’s investigational new drug (IND) application for Synacthen Depot to treat Duchenne muscular dystrophy (DMD). The invesigational drug is a depot formulation (a sustained-action drug formulation that allows slow release and gradual absorption) of Synacthen (tetracosactide), a synthetic…

Santhera Pharmaceuticals announced that it has received a $246,000 grant from the U.S. Food and Drug Administration (FDA) to support its ongoing Phase 1 clinical trial evaluating omigapil as a potential treatment for children and adolescents with congenital muscular dystrophy (CMD). The study, called CALLISTO (NCT01805024), is assessing the pharmacokinetics, safety, and tolerability of omigapil in…

Idera Pharmaceuticals recently presented pre-clinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentation, “Precise excision of targeted RNA by third generation antisense (3GA) oligonucleotides” was made at the…

Summit Therapeutics reports that a new formulation of ezutromid (called F6), its Duchenne muscular dystrophy (DMD) treatment candidate, achieved a greater than six-fold increase in maximum plasma levels in DMD patients participating in a Phase 1 clinical trial, compared to results achieved with its current formulation (F3), and at a lower…

Replenishing levels of a molecule naturally occurring in the body called NAD+ could restore muscle function according to scientists at the University of Pennsylvania.

Invitae, which specializes in genetic information, has expanded its neurology and cardiology test offerings by adding 11 new panels for genetic diseases. The company has also updated 17 of its neurology panels and eight of its cardiology panels based on recent discoveries in the field of genetics, including tests for muscular dystrophies.

Tivorsan’s lead investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (U.S. FDA). The human recombinant Biglycan (TVN-102), a natural component of muscles, activates a utrophin-based compensatory pathway that can potentially overcome damage caused by the…