In this issue: Acceleron trial of ACE-031 in DMD * Santhera trial of idebenone in DMD * NIH seeking parents’ perceptions of DMD/BMD ataluren trials * Becker MD study seeks participants * Biobank collecting blood samples for neuromuscular disease research * Taiwanese trial finds hydroxyurea ineffective in types 2 and 3 SMA
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Another trial of idebenone shows that, although safe and well tolerated, the substance doesn't lead to significant improvement in Friedreich's ataxia
Idebenone, a substance believed to act as an antioxidant and to aid in energy production in cellular structures called mitochondria, has failed to benefit people with Friedreich's ataxia (FA) in a phase 3 trial conducted at several European centers. The drug is similar to coenzyme Q10, a naturally occurring molecule.
Idebenone, an antioxidant and possible cellular energy booster, will be tested in some 240 boys with DMD
This story was updated 11/9/10 with contact information for this trial and information about clinical trials in general.
Pediatric Neurologist Richard Finkel, co-director of the MDA neuromuscular disease clinic at Children's Hospital of Philadelphia, will be the lead investigator for the North American arm of a large international trial with idebenone in Duchenne muscular dystrophy (DMD).
The following article contains items about: Friedreich's ataxia, Charcot-Marie-Tooth disease, myotonic muscular dystrophy type 1, amyotrophic lateral sclerosis, Emery-Dreifuss muscular dystrophy and distal muscular dystrophy (Miyoshi myopathy)
New MDA clinical research network to target ALS, DMD
Items in this article refer to clinical trials in: Duchenne muscular dystrophy, myasthenia gravis, Becker muscular dystrophy, limb-girdle muscular dystrophy, hypokalemic and hyperkalemic periodic paralysis, Pompe disease, FSH muscular dystrophy, Miyoshi distal muscular dystrophy, myotubular myopathy, congenital muscular dystrophy and ALS.