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 The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary […]
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. Genetic studies of families with a history of SMA allowed researchers to localize the region containing the gene responsible for SMA to the long arm of chromosome 5 in 1992.
Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene Read More »
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. This update covers a session on clinical research and outcome measures. The goal of this session is to present the results of important studies that could influence clinical trial design
2016 SMA Researcher Meeting Summary: Clinical Research Studies Read More »
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 with other clinical trials—we know that many in our community have questions. We have reached a new stage in our push for an FDA-approved treatment
Cure SMA Plans a Series of Educational Modules for the SMA Community Read More »
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 University of Missouri-based team is investigating ways to target SMN2, the SMA “backup gene.” Because of a genetic mutation in the SMN1 gene, individuals with
Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy Read More »
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 data also showed continued motor function improvement. AveXis has requested a Type B Meeting with the FDA to discuss SMA Type 1 clinical development pathway.
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 is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary
2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA Read More »
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 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
Novartis Releases Community Update on the Study of LMI070 Read More »
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 muscle weakness and/or muscle fatigue. CK 2127107 is liquid form taken twice a day by mouth. This first clinical trial of this investigational drug in
Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy Read More »
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 an important day in our collective pursuit of a meaningful treatment for Spinal Muscular Atrophy (SMA). We are pleased to share the exciting news that
