Front Page News
Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
During the 2016 SMA Researcher Meeting, the anniversary of the identification of the SMN genes by Judith Melki MD, PhD (Inserm, Universite Paris) was recognized. […]
Read More ›2016 SMA Researcher Meeting Summary: Clinical Research Studies
We will be posting a series of summaries from our 2016 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›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 […]
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 […]
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 […]
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. […]
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 […]
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 […]
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 […]
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 […]
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