AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy o...
AVEXIS (AVXS)
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Source: GlobeNewswire
– A mean increase in CHOP-INTEND of 17.3 was observed at three months post gene therapy in SMA Type 1 U.S. pivotal trial – – All patients in the therapeutic dose cohort in the Phase 1 trial were alive and event-free at 24-month follow-up – – Patients observed in long-term follow-up from the Phase 1 trial continued to gain strength and achieve new milestones, including two additional patients who could stand with assistance – CHICAGO, April 24, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will present initial results from the Type 1 U.S. Pivotal trial (STR1VE) and 24-month follow-up data from the Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1 at the 2018 Annual Meeting of the American Academy of Neurology (AAN) Annual Meeting in Los Angeles. “With a population closely matched to the Phase 1 tr
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