omigapil

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)

Targeting multiple pathways in mice with a disorder resembling merosin-deficient congenital muscular dystrophy shows more promise than aiming at one pathway at a time

posted on July 11, 2013 - 11:15am
Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.

Insights from pediatric neurologist Carsten Bönnemann

posted on July 1, 2013 - 9:23am
Quest Vol. 20, No. 3
Update (March 24, 2015): A phase 1 trial of omigapil in children and adolescents 5-16 years old who have merosin-deficient congenital muscular dystrophy and meet other study criteria is open in Bethesda, Md. See Assessment of Safety and Tolerability of Omigapil (CALLISTO), or enter NCT01805024 in the search box at ClinicalTrials.gov.
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.