Dyne Therapeutics’ Duchenne muscular dystrophy (DMD) therapy z-rostudirsen significantly improved dystrophin level and function versus placebo in boys aged four to 16 years with the condition who were eligible for exon 51 skipping therapy.

The results showed an increase in dystrophin levels to 5.46% at six months after adjusting for muscle content. This amounted to an approximate seven-fold change from baseline, which was around the same increase seen in earlier trials of z-rostudirsen.

DMD is a rare, progressive neuromuscular disorder caused by a range of different mutations in the DMD gene found on the X chromosome that cause loss of functional dystrophin protein. Around 12,000 individuals in the U.S. are affected by the condition and up to 2,200 of these are eligible for exon 51 s

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