The Summer 2016 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced $704,000 in funding for four new drug discovery grants. Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor neuron 1 (SMN1) […]
Summer 2016 Issue of Compass Now Available Read More »
We’re thrilled to announce a new partnership that will bring Cure SMA’s resources in front of The Mighty‘s wide-reaching readership. We will now have a growing home page on The Mighty and appear on many stories on the site, allowing us to get many more people involved with our organization. The Mighty is a story-based
We’re Partnering With The Mighty! Read More »
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 The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research.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
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 The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research.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 »
