Muscular Dystrophies

Funding from MDA and three other organizations will allow ReveraGen BioPharma to test its experimental anti-inflammatory drug for Duchenne MD in healthy volunteers

posted on July 17, 2014 - 8:00am
ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.

Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations

posted on July 10, 2014 - 1:59pm
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.

An experimental drug designed to reduce scarring in Duchenne MD-affected muscles shows signs of safety and desired activity; FDA will speed its review

posted on July 3, 2014 - 12:52pm
HT-100, an experimental drug being developed for Duchenne muscular dystrophy (DMD) with support from MDA, has shown preliminary safety and signs of the desired effects on scar tissue formation in the first 17 trial participants, and it has received "fast track" designation from the U.S. Food and Drug Administration (FDA).

A trial of an experimental compound to counteract muscle inflammation and scarring in Duchenne MD resumes, after an interruption to complete a dog safety study

posted on June 17, 2014 - 12:38pm
Update (July 3, 2014): Akashi says the HT-100 main trial is now closed; the extension study is open to those who have already participated in the main trial.

The European Medicines Agency expressed a 'positive opinion' on conditional approval for ataluren (Translarna) to treat Duchenne MD; a decision is expected within three months

posted on May 23, 2014 - 3:24pm
The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.

Access to eteplirsen may come in 2015; the company will conduct a confirmatory trial of eteplirsen and two additional trials of the drug

posted on May 19, 2014 - 2:33pm
Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S.

A pilot trial has found that one dose of tadalafil or sildenafil increased blood flow to Duchenne MD-affected muscles; a large tadalafil trial recently opened

posted on May 8, 2014 - 10:37am
A study conducted in 10 boys with Duchenne muscular dystrophy (DMD) has found that blood flow to exercising muscles is deficient and that treatment with either tadalafil (Cialis) or sildenafil (Viagra) normalizes this blood flow, at least in the short term (after one dose of either drug). Tadalafil and sildenafil, both PDE5 inhibitors, are approved by the U.S. Food and Drug Administration (FDA)...

The company says it hopes to have feedback from regulators about its experimental Duchenne MD drug drisapersen by the end of June 2014

posted on April 29, 2014 - 12:49pm
Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.