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
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
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.
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.