AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3
AVEXIS (AVXS)
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Source: GlobeNewswire
CHICAGO, April 25, 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, today announced that the first patient has been dosed in a Phase 3 trial evaluating AVXS-101 in pre-symptomatic patients with spinal muscular atrophy (SMA) Types 1, 2 and 3 (SPRINT). “Treating SMA as early as possible is critically important in order to rescue motor neurons before they are permanently lost. SPRINT enables us to understand how intervening in pre-symptomatic infants with AVXS-101 may impact clinical outcomes, including milestone development such as functional sitting, standing without support and walking,” said Dr. Sukumar Nagendran, Chief Medical Officer of AveXis. “In addition to our ongoing studies in SMA Types 1 and 2, SPRINT adds to our evaluation of AVXS-101 in multiple SMA sub-types, including Type 3. We are excited by the progress made across our
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