Registration for the Annual SMA Conference is well on its way to a record-setting number. Over 700 families and researchers have registered for the conference thus far—the highest number to this point in over five years! We are excited to have so many new and familiar faces joining us in June. If you have not […]
2016 Annual SMA Conference on Track to Break Attendance Records Read More »
Cure SMA has awarded a $140,000 research grant to Rashmi Kothary, MD, at the Ottawa Hospital Research Institute for his project, ” Muscle satellite cell biology and muscle regeneration in Smn-depleted mice.” Because of a genetic mutation, individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels. We know that
Cure SMA Awards $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute Read More »
Cure SMA has awarded a $140,000 research grant to Megerditch Kiledjian, PhD, at Rutgers, The State University of New Jersey, for his project, “Novel Strategies to Increase SMN2 RNA.” Because of a genetic mutation in the SMN1 gene, individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels. All individuals
Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers Read More »
The Senate Health, Education, Labor and Pensions (HELP) Committee has passed 7 bills out of committee as part of its “step-by-step” approach to Innovation and 21st Century Cures. Among them is The Advancing Research for Neurological Diseases Act of 2015 (S.849), sponsored by Sens. Isakson (R-Ga.) and Murphy (D-Conn.), which would expand research on neurological
Senate HELP Committee Passes Innovation Bills Read More »
Cure SMA has been carefully monitoring the consideration of 21st Century Cures on Capitol Hill. As you know, the House passed legislation in July last year, but the Senate has not acted on the bill or introduced their own version of “Innovation” legislation. Senator Alexander (R-TN), Chairman of the Senate Health, Education, Labor & Pensions
Update on 21st Century Cures/Innovation: the Senate’s “Step-by-Step” Approach Read More »
Cure SMA-funded researcher Stephen Kolb has published a paper in the journal Annals of Clinical and Translational Neurology. This paper reported on the baseline results from an ongoing biomarker and natural history study comparing infants affected by SMA to healthy infants of the same age. Research suggests that treatment for SMA may be more successful
Cure SMA-Funded Researcher Publishes Paper on Biomarker Study Read More »
The Prescription Drug User Fee Act (PDUFA), the law that allows the FDA to collect fees from drug manufacturers to fund the new drug approval process, is set to expire in September 2017. As part of the process to reauthorize this law, the FDA must hold monthly meetings with a stakeholders, patient groups, health care
Cure SMA Testifies Before FDA at PDUFA Meeting Read More »
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 »
