Shortly after of the announcement by Biogen and IONIS Pharmaceuticals of positive interim results from the Phase III clinical trial of nusinersen in spinal muscular atrophy (SMA, see Sep 2016 news), gene therapy company AveXis reported promising interim Phase I Trial results of their candidate SMA therapy, AVXS-101. While nusinersen increases expression of the SMN2 gene to compensate for lack of SMN protein, AVXS-101 is designed to deliver a functional copy of the mutated SMN1 gene (see Dec 2015 news). At a conference this month, the study principal investigator, Jerry Mendell of the The Research Institute at Nationwide Children’s Hospital in Ohio, reported that as of last month, 11 of 12 patients in the high dose cohort achieved head control and could sit unsupported. Remarkably, two patients have learned to walk independently. Detailed results, as well as adverse effects, were reported in the press release.
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