Santhera Pharmaceuticals is testing anti-cell death compound omigapil in children and teens with merosin-deficient and Ullrich congenital muscular dystrophies

posted on March 26, 2015 - 6:57pm
Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD)
posted on March 31, 2011 - 9:55am
QUEST Vol. 18, No. 2
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 For current information on clinical trials and studies, see the database at...

Another trial of idebenone shows that, although safe and well tolerated, the substance doesn't lead to significant improvement in Friedreich's ataxia

posted on May 28, 2010 - 12:09pm
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

posted on October 8, 2009 - 10:27am
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).
posted on October 1, 2009 - 1:53pm
QUEST Vol. 16, No. 4
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)
posted on November 1, 2008 - 1:57pm
QUEST Vol. 15, No. 6
New MDA clinical research network to target ALS, DMD
posted on November 1, 2007 - 1:12pm
QUEST Vol. 14, No. 6
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.