SMA Press
Update on LMI070 for the Treatment of Type 1 SMA
In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because results from an animal study, using daily dosing for a year compared to weekly dosing in the human study, showed unexpected…
Read MoreAveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for SMA Type 1
Chicago,Ill., July 20, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1…
Read MoreLetter to the SMA Community on the Study of LMI070 for the Treatment of Type 1 SMA
Wednesday May 25th, 2016 – Letter from Novartis: We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial, which is a common practice when studying potentially life threatening diseases to…
Read MoreFightSMA Announces First-Time Inclusion of Spinal Muscular Atrophy in Defense Department’s Peer-Reviewed Medical Research Program
WASHINGTON, D.C., May 26, 2016–FightSMA is proud to announce that, for the first time, the U.S. Senate Appropriations committee has included spinal muscular atrophy as a research topic in the Defense Department’s Peer-Reviewed Medical Research Program as part of the Fiscal Year 2017 Defense Appropriations bill. This exciting news comes as the result of months…
Read MoreSpinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development
-Phase 1 Study Initiated in Healthy Volunteers with New SMN2 Splicing Modifier – RG7916 – SOUTH PLAINFIELD, N.J., Jan. 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has…
Read MoreData from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress
Brighton, United Kingdom. October 5, 2015 – AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-101, the company’s initial product candidate for the treatment of spinal muscular atrophy (SMA) Type 1, at the International Congress of the…
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