Quest News (online only)

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...

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

PTC Therapeutics has begun submitting an application for U.S. approval of Translarna (ataluren) for Duchenne MD; completion of the application is slated for late 2015

posted on December 23, 2014 - 11:20am
New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy

posted on December 22, 2014 - 5:12pm
A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.

Pfizer will test its experimental drug PF-06252616 in ambulatory boys with Duchenne MD at multiple sites in the U.S. and elsewhere

posted on December 22, 2014 - 3:52pm
Multinational pharmaceutical comany Pfizer recently opened a phase 2 study of an experimental compound that may be beneficial in Duchenne muscular dystrophy (DMD) through its ability to block myostatin, a naturally occurring protein that is known to limit muscle growth.

MDA-supported Summit PLC announced it will continue testing it experimental drug in Duchenne MD patients, along with dietary modifications that may improve drug absorption

posted on December 22, 2014 - 11:22am
SMT C1100, an experimental compound designed to boost levels of the potentially therapeutic protein utrophin in patients with Duchenne muscular dystrophy (DMD), will undergo further testing in boys with this disorder along with dietary specifications that may improve its absorption by the body, its developer has announced.

In this second of two fall reports on treatment development for Duchenne muscular dystrophy, news about ARM210, CAP-1002, PDE5 inhibitors, drisapersen and CAT-1004 is presented

posted on December 4, 2014 - 3:13pm
Several experimental drugs are in development to treat Duchenne muscular dystrophy (DMD), a genetic disorder that results in a lack of the dystrophin protein in cardiac and skeletal muscle cells.