PR NewswireData From ISIS-SMN Rx Phase 1 Study in Children With...

PR Newswire
Data From ISIS-SMN Rx Phase 1 Study in Children With Spinal Muscular Atrophy Presented at the American Academy of Neurology Meeting

March 20, 2013 7:13 PM ET

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TICKERS IN THIS ARTICLE
NAME LAST CHNG % CHNG
ISIS 17.84 +0.28 +1.59
ISIS
SMA 11.24 +0.14 +1.26
AAN 28.87 +0.32 +1.12
SMN 49.30 -0.59 -1.18

Isis to host an investor event and live webcast at 7:30 am PT on Thursday, March 21 in San Diego

CARLSBAD, Calif., March 20, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. ISIS announced that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA +1.26%, news) were presented today at the 65th American Academy of Neurology (AAN +1.12%, news) Annual Meeting by Dr. Claudia Chiriboga, from Columbia University Medical Center. In the presentation, Dr. Chiriboga reported that, in this single-dose, open-label study, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and that improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA.