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.
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.
Recently published findings from two independent groups have suggested possible treatment pathways for the merosin-deficient and integrin-deficient forms of congenital muscular dystrophy (CMD).
Doxycycline fights cell death and lessens disease severity in merosin-deficient mice