Study of the efficacy of drugs with the capacity to restore the expression of dystrophin in myoblasts in patients affected by Duchenne Muscular Dystrophy with Nonsense mutations. Analysis of the causes for variability of response and assessment of strategies to increase the efficacy in human myoblasts and in mdx mice.
HUVH: University Hospital Vall d’Hebrón, Barcelona (Spain)
VHIR: Vall d’Hebrón Institute of Research, Barcelona (Spain)
Investigatorsr: Dr. Francina Munell MD, Dr. Elena Martínez Sáez MD, Dr. Jordi Barquinero MD, Dr. María José Pérez García MD, Dr. Salvador Borrós MD, Dr. David Sánchez García MD and Dr. Ofir Arad MD.