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news.GlaxoSmithKline (GSK) and Prosensa have treated first patient in GSK2402968 Phase III trial in ambulant boys with duchenne muscular dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD).
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GSK2402968, an antisense oligonucleotide which induces exon skipping of exon 51, is currently in late stage development for DMD.
The study which is designed to evaluate the safety and efficacy of GSK2402968 6mg/kg, in ambulant boys over 5 years of age with DMD, is expected to recruit around 180 patients.
GSK Rare Diseases chief medical officer and Development head Philippe Monteyne said the commencement of this Phase III study is an important milestone.
Prosensa chief medical officer Giles Campion said if the results of this study are positive, they hope it will lead to an approved treatment option for the thousands of young people worldwide living with this devastating disease.