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 »
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 »
