Cure SMA would like to thank the Fighting for Kaiden Foundation for their generous donations to Cure SMA programs. That generosity is providing both support for today and hope for the future to those affected by SMA. The foundation recently donated 15 Leckey Advanced Bath Chairs, two EASyS Strollers, and two large boxes of toys […]
Cure SMA Receives Generous Donation from Fighting for Kaiden Foundation Read More »
Biogen and Ionis Pharmaceuticals recently provided an update on the clinical development of nusinersen (IONIS-SMNRx), which is currently being tested in Phase 3 clinical trials: Dear members of the SMA community, As we begin 2016, we wanted to provide an update on the nusinersen (IONIS-SMNRX) program to you and the many families around the world
Biogen and Ionis Provide Update on Nusinersen (IONIS-SMNRx) Read More »
Abstract submission is now open for the 2016 SMA Researcher Meeting, which will be held June 16-18 as part of the Annual SMA Conference in Anaheim, CA. Interested researchers may visit our abstract submission form for more information and instructions. The deadline to submit an abstract is Friday, March 4. Addressing the Most Vital Questions
Abstract Submission Opens for 2016 SMA Researcher Meeting Read More »
Roche, along with partners PTC Therapeutics and the SMA Foundation, recently provided the following update on the development of RG7800 and on a new clinical trial planned for RG7916: “Dear SMA community, “The collaboration between Roche, PTC Therapeutics and the SMA Foundation would like to provide you with an update on the advance of our
Roche Announces New Clinical Trial of RG7916 Read More »
AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy for SMA. The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined
AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial Read More »
Cytokinetics has opened enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV. We are excited to see the continued progress of this drug into the next phase of clinical trials, and particularly excited to see this trial focused on teens and adults. This latest trial
Cytokinetics Opens Enrollment for Phase 2 Trial Read More »
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use
Cure SMA Funding Leads to 18 New Research Publications in 2015 Read More »
Our approach to research is focused on continually expanding the pipeline of potential drugs and therapies for SMA, cultivating new approaches and advancing promising ideas. Every year, the pipeline gets broader and deeper. We’ve seen enormous growth over the past decade, and we expect that growth to continue in the coming years as more projects
Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs Read More »
Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives. Our programs provide tangible support for families affected by SMA, easing the difficulties of an SMA diagnosis, and showing each family that they are not alone and that there is hope. Our family
Cure SMA Reaches Thousands of Families in 2015 Read More »
On December 1, more than 120 people from the SMA community—including government, and industry partners—gathered for the 5th Annual “Hope on the Hill” Congressional Dinner. Several Members of Congress attended the dinner, including Speaker of the House Paul Ryan, Congressman Sean Duffy, Congressman Richard Hudson, Congressman Bill Huizenga, Congressman Erik Paulsen, Congressman David Schweikert, and
5th Annual Congressional Dinner Brings Together Families, Government and Industry Read More »
