Duchenne Muscular Dystrophy (DMD)

UPDATE: DMD: Eteplirsen Shows Continued Benefit at 84 Weeks

DMD: Experimental Exon-Skipping Drug Drisapersen Disappoints

A large-scale, phase 3 trial of the experimental exon-skipping drug drisapersen, in development to treat Duchenne muscular dystrophy (DMD), found no statistically significant differences on tests of walking distance or motor function between trial participants treated with the drug and those treated with a placebo.

'Gentler' Corticosteroid Drug Shows Promise in DMD Mice

A drug that may provide the benefits of corticosteroid medications such as prednisone and deflazacort without some of their notorious side effects — growth retardation, bone loss and suppression of the immune system — has been shown to protect and strengthen muscles in mice with a disease that mimics Duchenne muscular dystrophy (DMD).

DMD: Summit Granted Patents for Utrophin Booster

Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it now has two U.S. patents for its experimental drug SMT C1100 for Duchenne muscular dystrophy (DMD).

The patents will provide commercial protection to the company as it continues the further development of this drug. However, the drug will not become available to patients until it receives approval from regulatory agencies.

DMD: Prosensa Announces New Grant and Provides Exon-Skipping Update

Dutch biotechnology company Prosensa, developer of experimental drugs to treat Duchenne muscular dystrophy (DMD) using exon skipping, announced encouraging developments in an Aug. 28, 2013, press release.

Fayetteville, N.Y., Twins with Duchenne Muscular Dystrophy To Be Featured on National MDA Telethon

MDA's 'Bridge-to-Industry' Program Awards Second Grant

Christopher Penton recently completed his doctorate (Ph.D.) in integrated biomedical sciences at Ohio State University in Columbus.

MDA Awards $8.5 Million to 31 Neuromuscular Disease Research Projects

In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.

“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform' technologies, where successes can be transferred well beyond the specific disease in which they are developed and tested.”

DMD: Exon-Skipping Drug Drisapersen Appears to Increase Dystrophin Levels

Multinational pharmaceutical company GlaxoSmithKline (GSK) has released encouraging results about dystrophin protein production in a phase 2, non-U.S. trial of its exon-skipping compound drisapersen in boys with Duchenne muscular dystrophy (DMD).

MDA Funds Study of Prednisone in Very Young Boys with DMD

Pediatric neurologist Anne Connolly has received an MDA grant to study the effects of prednisone on Duchenne muscular dystrophy in very young children.

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