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Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its primary endpoint, which was to assess the safety and tolerability of ATL1102 in patients with DMD who are non-ambulatory (unable to walk independently).
After six months of weekly, subcutaneous treatment with a low dose, the drug appeared to be safe and well tolerated in all participants with only minor side effects. Secondary endpoint results also seemed encouraging, as the drug lowered the average number of specific immune cells during the treatment period and largely improved measurements of functional capacity in seven of nine boys treated.
DMD is a genetic disease caused by a mutation in the dystrophin gene on the X chromosome that results in little or no production of dystrophin, an essential protein for keeping muscle cells intact.
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