The company plans to submit a New Drug Application to the U.S. Food and Drug Administration later this year, and has committed to conducting two confirmatory post-approval clinical trials

posted on June 6, 2014 - 9:56am
Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.

In a September 2013 presentation, Prosensa CEO Hans Schikan said the company will continue developing exon-skipping drugs for Duchenne MD, despite disappointing drisapersen results

posted on September 27, 2013 - 12:37pm
"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.

MDA and AFM co-sponsored a symposium on neuromuscular gene therapy at the 2012 meeting of the American Society for Gene & Cell Therapy

posted on May 22, 2012 - 2:25pm
Overcoming barriers to gene therapy — the delivery of therapeutic genes — to treat neuromuscular diseases was the topic of a symposium jointly sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 15th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).

Clinical trials of two different exon-skipping compounds show encouraging results; Duchenne MD participants are being sought for new trials

posted on March 29, 2011 - 11:42am
sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in the United States and elsewhere. Exon skipping for DMD is a strategy that coaxes muscle fibers to ignore, or "skip," the genetic instructions for certain parts of the dystrophin gene so that functional...

Researchers have shown that the protein interleukin 10 reduces inflammation and improves muscle repair in a mouse model of Duchenne muscular dystrophy

posted on March 9, 2011 - 2:32pm
A team of researchers at the David Geffen School of Medicine at the University of California-Los Angeles (UCLA) has demonstrated that the naturally occurring protein interleukin 10 (IL10) may help reduce harmful inflammation and promote muscle regeneration in people with Duchenne muscular dystrophy (DMD), and potentially those with other forms of muscular dystrophy. MDA supported James Tidball,...

The National Institutes of Health has awarded more than $4.5 million to specialized muscular dystrophy research centers at three U.S. institutions

posted on September 30, 2010 - 3:47pm
The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy. Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University...

News on Duchenne muscular dystrophy, Friedreich's ataxia, autoimmune diseases, and cell and gene therapies

posted on September 24, 2010 - 1:39pm
Duchenne muscular dystrophy

The experimental drug ataluren, developed to overcome nonsense mutations in Duchenne and Becker MD, did not meet its primary end point in a large-scale human trial

posted on March 3, 2010 - 4:55pm
The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well tolerated, failed to meet its primary end point within the 48-week duration of the phase 2b trial. That end point was an improvement in how far boys with DMD or BMD could walk in six minutes.