gene replacement therapy

Insights from pediatric neurologist Carsten Bönnemann

posted on July 1, 2013 - 9:23am
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 - 9:22am
QUEST Vol. 14, No. 6
Items im this article refer to research in:  Duchenne muscular dystrophy, myotonic muscular dystrophy, facioscapulohumeral muscular dystrophy and spinal muscular atrophy.