exon 51

Boys with DMD continuously treated with eteplirsen for three years walked farther in six minutes than those receiving a placebo for the first six months

posted on January 13, 2015 - 3:36pm
Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of...

A phase 2 trial of Duchenne MD drug drisapersen found the high-dose group walked farther than the placebo group at 24 and 48 weeks

posted on March 18, 2014 - 4:10pm
Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014. The approximately one-hour presentation is archived at Events & Presentations on the Prosensa site and includes a thorough discussion of Prosensa's plans for drisapersen and other exon-...

Dutch biopharmaceutical company Prosensa outlined plans for drisapersen and other drugs for Duchenne MD in a Jan. 16, 2014, conference presentation and press release

posted on January 17, 2014 - 3:53pm
Update (Jan. 21, 2014): Both the Jan. 16, 2014, webcast from the J.P. Morgan conference and the Jan. 21 webcast for patient groups are available on the Prosensa site at Events & Presentations. The patient group webcast recaps many of the same themes as those summarized below from the J.P. Morgan conference presentation.

An increase in dystrophin was seen in a foot muscle in 61 percent of boys with nonsense-mutation DMD treated with ataluren in an MDA-supported study

posted on January 15, 2014 - 9:47am
An increase in levels of the dystrophin protein was seen after treatment with the experimental drug ataluren (originally called PTC124) in an MDA-supported, phase 2a, open-label trial of 38 boys with Duchenne muscular dystrophy (DMD) caused by a specific type of mutation in the dystrophin gene.

The FDA will speed up the review process for a promising exon-skipping drug designed to treat Duchenne MD caused by specific dystrophin mutations

posted on July 3, 2013 - 9:51am
Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

Weekly injections of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy resulted in a significant difference in walking distance compared to a placebo

posted on April 11, 2013 - 11:59am

10 boys with Duchenne muscular dystrophy continue to show stabilization of walking ability; further trial results will be announced at an MDA conference in late April

posted on April 5, 2013 - 11:08am
Eteplirsen, an experimental exon-skipping therapy designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 74 of a phase 2b, 12-person study.

The 62-week results from a phase 2b trial of eteplirsen show the drug continued to stabilize walking ability in boys with Duchenne MD

posted on December 7, 2012 - 4:57pm
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 weeks with respect to the distance walked in six minutes.