News

Australia’s drug regulatory agency, the Therapeutic Goods Administration (TGA), has granted  Santhera Pharmaceuticals’ lead product Raxone (idebenone) orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). Raxone (sold in some markets under the trade name Catena) has already received a similar designation from European, Swiss, and…

 PTC Therapeutics marked Sept. 7 — World Duchenne Awareness Day — by announcing the four winner of the company’s 2016 global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program. STRIVE awards are granted in recognition of extraordinary excellence and innovation by nonprofit organizations that serve rare disease communities, such as…

Alysia Vrailas-Mortimer and her research team at Illinois State University have received a $435,000 grant from the National Institutes of Health (NIH) to continue unveiling the genetics of muscular dystrophy, specifically “limb girdle” muscular dystrophy. The team studies this special form of muscular dystrophy, which attacks muscles around the shoulders…

The U.S. Food and Drug Administration (FDA) has allowed an investigational new drug (IND) application for Carmeseal-MD in Duchenne muscular dystrophy (DMD), according to Phrixus Pharmaceuticals. Carmeseal-MD (Poloxamer-188 NF, or P-188 NF) is the first disease-modifying agent in diseases characterized by membrane instability such as Duchenne MD or limb…

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…