Kay Davies

SMT C1100, which increases levels of the protein utrophin, reached adequate blood levels in healthy volunteers; trials in Duchenne MD may be next

posted on October 10, 2012 - 4:15pm

Exon skipping proves effective in mice with a severe DMD-like disease

posted on October 23, 2009 - 10:11am
Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have. Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and...