Latest News
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 […]
Read More ›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 […]
Read More ›Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
Biogen and Ionis have been testing nusinersen in clinical trials and today announced that they will close ENDEAR, the Phase 3 trial testing nusinersen in infants with SMA type 1. […]
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