Clinical Trial, BeNeLux
Prosensa Publishes DMD study results in NEJM
Prosensa NV and researchers from Leiden University Medical Center (LUMC) under Prosensa's scientific advisor Prof. Gertjan van Ommen have published results from a Phase I clinical study with the 2-’O-methyl antisense compound PRO051 in four children (aged 10-13) with Duchenne Muscular Dystrophy (DMD) (N Engl J Med. 357(26):2677-86). Intramuscular injection of a single 80 mg shot of the compound, which is able to correct the open reading frame of the DMD gene by exon skipping and thus to restore dystrophin expression, was not associated with clinical adverse reactions, according to Prosensa. Additionally, PRO051 restored dystrophin expression in the treated muscle fibers of all four patients, who had been selected on the basis of their mutational status, muscle condition, and positive exon-skipping response to PRO051 in vitro. The compound will be suitable for 13% of all DMD patients (DMD incidence
1: 3,500), because it can treat a cluster of DMD mutations.