Participate in Annual Cure SMA Community Update Survey – Open Now!

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Last year presented the us with unique challenges, some we are still managing today. However, it has also reinforced the importance of understanding what the SMA community is thinking, feeling, and experiencing in their day-to-day. Knowing our community’s needs allows us to better serve you—whether it be advocating for comprehensive research, educating regulators, payers, and […]

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Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry

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Cure SMA’s primary focus is supporting care of the highest value to individuals with SMA. As a result of treatments approved by the U.S. Food and Drug Administration (FDA), as well as successful initiatives like the Cure SMA Care Center Network and SMA Clinical Data Registry, we have made significant progress enhancing the lives of

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Cure SMA Launches LifeVac Support Program to Help in Choking Event

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Along with funding SMA research and care, Cure SMA is proud to provide thousands of individuals with SMA and their families with vital support they need to navigate life with SMA. In an effort to always be offering additional resources, we are thrilled to launch a new program for all individuals with SMA to help

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Scholar Rock Community Statement on TOPAZ 12-Month Topline Data

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Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release.  Dear Members of the SMA Community, We are pleased to share 12-month topline results from our TOPAZ study, a Phase 2 clinical trial to evaluate the safety and efficacy of apitegromab (SRK-015) in individuals with

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Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on SMA-HI, a Clinical Trial Outcome in Adults with SMA

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Dr. Chad Heatwole, a Cure SMA-funded researcher, and his team have published a paper in the Journal, Muscle Nerve, titled “The Spinal Muscular Atrophy Health Index (SMA-HI): A Novel Outcome for Measuring How a Patient Feels and Functions.” This project, funded by Cure SMA, focused on developing a multi-faceted, disease-specific, patient-reported outcome to measure an SMA

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Nusinersen Research Updates Presented by Biogen

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Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND clinical trials. Oral presentation details: Title: Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies Presenter: Laurent Servais, MD, PhD Title: Nusinersen in Children with SMA Who

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Research Updates from Scholar Rock

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Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in SMA Scholar Rock recently shared 6-month interim analysis data from the TOPAZ Phase 2 clinical trial evaluating the potential of apitegromab, a highly selective inhibitor of latent myostatin, in improving motor function in patients with Types 2 and 3 spinal muscular atrophy (SMA). A this

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Community Statement from Genentech

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Dear SMA Community, Based on our commitment to the SMA community to provide transparent, timely updates related to our medicines, we are writing to share information regarding recent reports of leaking bottles of Evrysdi, and provide information on steps we are initiating as a result. Below we have provided additional details and next steps should

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Community Statement from Novartis Gene Therapies

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Dear Cure SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of ZOLGENSMA® (onasemnogene abeparvovec-xioi). As with all commercially marketed products, we are continuously monitoring the safety of ZOLGENSMA, and sharing safety data as part of the required pharmacovigilance with health

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