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
"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.
Clinical trials of two different exon-skipping compounds show encouraging results; Duchenne MD participants are being sought for new trials
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
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,...
News about research in congenital myasthenic syndromes, Duchenne and limb-girdle muscular dystrophies, Pompe disease and induced pluripotent stem cells
Congenital myasthenic syndromes
A multinational team of scientists has identified mutations in the gene for glutamine-fructose-6-phosphate transaminase 1 (GFPT1) as responsible for some forms of a congenital myasthenic syndrome (CMS).
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
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.
PTC Therapeutics, with support from MDA, is testing ataluren in boys with DMD or BMD who have nonsense mutations and are no longer walking.
Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted.
Ms. Wheelchair Michigan spreads message of self-advocacy and inclusion