June 2016

Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials

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At the Annual SMA Conference, representatives from the six programs currently in clinical trials gave an update on their programs. Of these six programs, four— AVXS-101 (gene therapy), nusinersen, RG7800/RG7916, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to replace the mutated in the SMN1 gene that causes SMA, and the other three […]

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Cure SMA Posts Keynote Presentations on the NDA Process

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The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and how that approval process works. As we get closer to

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Cure SMA Awards $304,000 Drug Discovery Grant to Livio Pellizzoni, PhD, at Columbia University

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Cure SMA has awarded a $304,000 drug discovery grant to Livio Pellizzoni, PhD, at Columbia University. The award is for the project, “Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA.” About the Project Dr. Pellizzoni and his colleagues at Northwestern University have identified a novel cellular pathway, called p38αMAPK, that is altered

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Cure SMA Announces Biogen as Presenting Sponsor for the 2016 Annual SMA Conference

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Cure SMA is pleased to announce that Biogen will be the Presenting Sponsor for our 2016 Annual SMA Conference. This year’s conference is set to be a significant event for the SMA community, with over 1,400 families and researchers coming together in Disneyland, June 16-19. “Without the generosity and support of Biogen, it would not

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Cure SMA Releases June 2016 Update to the SMA Drug Pipeline

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We’ve recently released an update to the SMA drug pipeline. This latest version includes: 18 active programs. 14 pharmaceutical partners. 6 programs in clinical trials. 28 programs in the cumulative pipeline total, including 10 failures to date. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of

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Thank you to Our 2016 Annual SMA Conference Exhibitors!

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Thank you to the following exhibitors who have generously supported our 2016 Annual SMA Conference. This one-of-a-kind event would not be possible without your support! For more information on any of these companies, please visit our conference exhibitor page. Conference Exhibitors Platinum Exhibitors Gold Exhibitors Gunnell, Inc. Innovation In Motion PromptCare RespirTechThomashilfen North America Triumph

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Biogen and Ionis Release Spring 2016 Community Update

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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 approach the upcoming Cure SMA conference in Anaheim, we want to provide an update on the status of the nusinersen program and

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