Clinical Trials and Studies

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...

Research updates and clinical trials information

posted on January 5, 2015 - 9:07am
Quest Winter 2015
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

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 a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site

posted on December 2, 2014 - 2:11pm
Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed  to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12

posted on November 21, 2014 - 10:19am
Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional...

The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)

posted on November 19, 2014 - 3:36pm
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.

Four of six men with Becker muscular dystrophy increased their six-minute walking distance after injections of follistatin genes into the thigh muscles of both legs

posted on November 19, 2014 - 1:05pm
Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

posted on November 5, 2014 - 4:03pm
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014. PTC is moving forward with ataluren