A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy
Neuromuscular Disorders Nov 05, 2017
Goemans N, et al. - The current phase 3 study (DEMAND III; DMD114044; NCT01254019) assessed the efficacy and safety of subcutaneous (sc) drisapersen 6 mg/kg/week over 48 weeks in a larger cohort of ambulant Duchenne muscular dystrophy (DMD) subjects. Collected evidence represented that in a less impaired population of DMD subjects, drisapersen appeared to be beneficial.
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