Research Updates

Santhera Pharmaceuticals' investigational drug to treat respiratory dysfunction in Duchenne muscular dystrophy has shown benefit compared to a placebo in a phase 3 trial

posted on May 29, 2014 - 10:23am
Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.

In a May 22, 2014, letter, Prosensa provides updates on drisapersen and other Duchenne MD compounds in its pipeline, and on its natural history study

posted on May 28, 2014 - 8:12am
Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.
posted on October 3, 2013 - 9:15am
Quest Vol. 20, No. 4
In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million. 
posted on October 3, 2013 - 9:01am
Quest Vol. 20, No. 4
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posted on July 1, 2013 - 9:04am
Quest Vol. 20, No. 3
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posted on April 1, 2013 - 9:03am
Quest Vol. 20, No. 2
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A follistatin gene therapy compound gains orphan drug status; potential improvements are reported for utrophin-based therapies, exon skipping and stem cell transplantation

posted on December 27, 2012 - 5:00am
Update (Jan. 23, 2013): The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri. Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.

The drug improved heart function and survival in mice with lamin A/C mutations, which cause lamin A/C-related Emery-Dreifuss MD and type 1B limb-girdle MD

posted on May 18, 2012 - 2:00pm
Update (June 1, 2012): This story was updated to include the availability of a podcast on this topic.