Sarepta Therapeutics has announced encouraging results on their PPMO drug candidate for individuals with Duchenne muscular dystrophy amenable to Exon 51 skipping. Preliminary data for the 30mg/kg dose shows 6.55% mean dystrophin expression and predicting longer-term use will achieve greater than 10% dystrophin production. Hypomagnesemia was identified as a safety concern, but all cases are now resolved and, with supplementation, is manageable. Sarepta will be moving forward with the 30mg/kg dose, and hopefully, they will resume the second part of their study soon at this new dose.
We want to thank all our families who have dedicated their time to this and all Duchenne trials. We appreciate the sacrifices made to participate in these clinical trials, and because of you, we are another step closer to finding a treatment for our boys.
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