First targeted treatment success for Duchenne Muscular Dystrophy

Prof Francesco Muntoni interview

Scientists at the UCL Institute of Child Health (ICH) have led on an important breakthrough in the development of a treatment for Duchenne muscular dystrophy, as detailed in The Lancet today. 

Together with the MDEX Consortium where the ICH's Prof Francesco Muntoni is the Chair, the group have shown that an antisense oligonucleotide genetic therapy was effective in restoring the missing protein dystrophin in 7/19 trial participants, with 3 in the higher dose cohort showing for the first time levels in dystrophin exceeding 18% of those found in normal muscle. Their laboratory findings suggest that this approach could provide a functional advantage to affected patients with eligible mutations.