Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers

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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 […]

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Senate HELP Committee Passes Innovation Bills

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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

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Update on 21st Century Cures/Innovation: the Senate’s “Step-by-Step” Approach

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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

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Cure SMA-Funded Researcher Publishes Paper on Biomarker Study

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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

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Cure SMA Receives Generous Donation from Fighting for Kaiden Foundation

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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

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Biogen and Ionis Provide Update on Nusinersen (IONIS-SMNRx)

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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

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Abstract Submission Opens for 2016 SMA Researcher Meeting

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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

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Roche Announces New Clinical Trial of RG7916

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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

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AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial

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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

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