Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers

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In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide $1.2 million to this Network of highly skilled clinical trial investigators, clinical evaluators, clinical coordinators, statisticians, and data management personnel. The PNCRN […]

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Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S.

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    Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, titled ‘The SMA Clinical Trial Readiness Program: Creation and Evaluation of a Program to Enhance SMA Trial Readiness in the United States.’ The Cure SMA Clinical Trial Readiness Program is

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Registration Now Open for the 2020 Virtual SMA Conference

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Cure SMA has released the agenda and registration details for the 2020 Virtual SMA Conference, which will be held from Monday, June 8th – Friday, June 12th. We greatly look forward to virtually uniting as a community in just a few short weeks! It is our #SMAConferenceAtHome. Check out the Virtual SMA Conference Booklet for

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Biogen Shares SPINRAZA Research Update

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New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the sustained efficacy and longer-term safety of SPINRAZA in a broad range of patients with spinal muscular atrophy (SMA). These new data were

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Cure SMA Announces a 2020 Virtual SMA Conference – United, At Home

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Cure SMA has been hosting the Annual SMA Conference since 1988, bringing together leading researchers, clinicians, affected individuals, and families living with SMA. The weekend has traditionally been filled with a wide variety of opportunities to learn about the latest in treatments, research, advocacy, care, and support. But most of all, it was a time

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Cure SMA Announces Summit of Strength Virtual Webinar Series

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For the past two years, Cure SMA’s Summit of Strength program has brought together thousands of SMA community members for the opportunity to network and learn about the latest advances in treatment, care, advocacy, and support. Considering event cancellations across the country due to the ongoing pandemic, Cure SMA is now offer these resources through

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Biogen Releases Q2 Community Statement on Latest Milestones in Clinical Development Program

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Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working with health systems, institutions, physicians, patients and their families to navigate potential SPINRAZA® (nusinersen) dosing delays related to COVID-19. The health and safety of patients is our primary concern, and

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Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA

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Scholar Rock recently reported financial results for the first quarter ending March 31, 2020, highlighting progress and upcoming milestones for its pipeline programs—include the SRK-015 program for spinal muscular atrophy (SMA). Scholar Rock plans to report interim efficacy, safety, pharmacokinetic (PK), and pharmacodynamic (PD) results in the fourth quarter of 2020 for patients across the

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Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA

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      Genentech, a member of the Roche Group, today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1-7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41) sitting without support for 5 seconds by month

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Annual Cure SMA Community Update Survey Ready for Your Input

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Navigating the recent global health crisis, Coronavirus (COVID-19), has reinforced for us the importance of understanding what the SMA community is thinking, feeling, and experiencing. This allows us to more effectively support those impacted by SMA, both with the everyday needs you have and during greater public health challenges. Without this knowledge, we cannot advocate

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