Latest News
Cure SMA Plans a Series of Educational Modules for the SMA Community
With the recent news that Biogen and Ionis will submit nusinersen for FDA approval—along with other recent developments, including the breakthrough designation for AveXis’s gene therapy program, and ongoing work […]
Read More ›Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
Dr. Chris Lorson and his colleagues have published a paper, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy,” in the journal Molecular Therapy. The […]
Read More ›AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
AveXis recently released new data from the Phase 1 clinical trial of AVXS-101, a gene therapy program to treat SMA. The interim data through July 1 showed no “events.” The […]
Read More ›2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting […]
Read More ›Novartis Releases Community Update on the Study of LMI070
Novartis recently provided a community update on clinical trials for LMI070. This information is effective as of July 25 2016: “In May, we shared with you news of the difficult […]
Read More ›Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy
CK-2127107 is a novel fast skeletal muscle troponin activator being developed as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular conditions associated with […]
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