Duchenne Muscular Dystrophy (DMD)

MDA Grant Will Help Families in DMD Steroid Study

Robert Griggs, a professor of neurology at the University of Rochester (N.Y.), has received an MDA grant of $237,316 over three years to support travel costs for North American participants in a large, multinational trial to determine which corticosteroid treatment regimen is best for children with Duchenne muscular dystrophy (DMD).

DMD — Robert Griggs, M.D.

Robert Griggs, a professor of neurology at the University of Rochester in New York state, received an MDA grant of $237,316 over three years to cover travel costs for North American participants in a clinical trial of prednisone and deflazacort, two corticosteroid drugs used to treat Duchenne muscular dystrophy (DMD).

DMD: Sarepta Updates Community on Eteplirsen Plans, Pipeline

Sarepta Therapeutics announced it is on track to submit an application to the U.S. Food and Drug Administration (FDA) for approval of eteplirsen in the first half of 2014.

In parallel with that application, the company is preparing to conduct a large-scale, confirmatory trial of eteplirsen. It plans to open the trial during the first quarter of 2014.

DMD: Companies To Host Web Presentations on Exon Skipping

Prosensa Says It Remains Committed to DMD

"Our commitment to Duchenne muscular dystrophy remains," said Hans Schikan, CEO of Netherlands-based biotechnology company Prosensa at a Sept. 27, 2013, presentation in New York.

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.

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