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
  Update (Feb. 6, 2015): This article has been amended to show that Catabasis' compound CAT-1004 is expected to move into clinical testing in boys with DMD during the first half of 2015. =======================================================================================================================

BioMarin has purchased the rights to Repligen’s histone deacetylase (HDAC) inhibitor portfolio, including the experimental FA drug RG2833, whose development MDA has supported

posted on January 22, 2014 - 3:01pm
Experimental Friedreich's ataxia (FA) drug RG2833, which has been in early-stage development by Massachusetts company Repligen with support from MDA, is now owned by California biotechnology company BioMarin.

Jacobus is inviting 30 selected individuals with Lambert-Eaton myasthenic syndrome to participate in a placebo-controlled trial of 3,4-DAP

posted on January 25, 2012 - 6:00am
Update (Oct.

Clinical trial testing experimental enzyme replacement drug in people with Pompe disease who are at least 13 years old

posted on November 8, 2011 - 10:08am
Update (March 27, 2013): BioMarin announced March 19, 2013, that it plans to move to a phase 2/3 trial of this drug for late-onset Pompe diseae, pending a review by regulatory authorities. See Pompe disease: Late-onset treatment moves forward.

Reports on research in amyotrophic lateral sclerosis, Duchenne muscular dystrophy, myotubular myopathy and spinal muscular atrophy

posted on August 12, 2010 - 11:33am
Duchenne muscular dystrophy Acceleron Pharma announced Aug. 4 that it has received fast track designation from the U.S. Food and Drug Administration (FDA) for its experimental compound ACE031 for the treatment of Duchenne muscular dystrophy (DMD). ACE031 is designed to interfere with the actions of myostatin, a protein that inhibits muscle growth.
posted on November 1, 2008 - 1:57pm
QUEST Vol. 15, No. 6
New MDA clinical research network to target ALS, DMD