Latest News
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 […]
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 […]
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 […]
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 […]
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 […]
Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings
Today, Biogen and Ionis Pharmaceuticals provided a statement regarding the decision to submit the first ever SMA regulatory filings for FDA approval. Dear Members of the SMA Community, Today marks […]