Record Number of Abstracts Submitted for the 2019 SMA Researcher Meeting

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Abstract submission is now closed for the 2019 SMA Researcher Meeting. We are excited to announce that we received 166 submissions, a record number. Our submissions come from researchers in 20 countries, 11 companies, and close to 65 institutions. We expect over 500 researchers and clinicians from almost 100 organizations will attend the SMA Researcher […]

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Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration

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Today, Cure SMA and Parent Project Muscular Dystrophy (PPMD) announced their strategic collaboration to collect real-world data to improve the lives of those affected by spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (Duchenne). Both prominent organizations have joined forces with Prometheus Research as their principal informatics partner, enabling unprecedented levels of collaboration on data

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Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment

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For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily life with SMA and the ongoing

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Recording of Genentech/Roche and Cure SMA Webinar Now Available Online

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A recording of this week’s webinar with Genentech/Roche is now available online. During the webinar, Genentech/Roche shared their history working with the SMA community, and their investigational medicine risdiplam. Updates on their clinical trials in pre-symptomatic & types 1, 2 & 3 were also shared. View the webinar  About Risdiplam Risdiplam is an investigational, oral

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Cure SMA Advocates for SMA Community at ICER Hearing

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On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). ICER is an independent health policy group that evaluates the effectiveness of drug treatments, and the costs associated with those treatments. The purpose of the meeting was to review both

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Biogen Provides 2019 Community Update on Spinraza

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Dear members of the SMA community, As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. In recent weeks and months, we engaged the community on a number of different initiatives and are excited to share updates on our activities, support of new clinical research and

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Genentech/Roche and Cure SMA to Co-Host Webinar

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On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the webinar, you’ll hear from Genentech/Roche on their history working with the SMA community, and their investigational medicine risdiplam. You’ll hear updates on their clinical trials in pre-symptomatic & Types 1,

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New Dates and Locations Announced for 2019 Summits of Strength

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Cure SMA is pleased to announce that we will be continuing the Summit of Strength Program in 2019, adding 20 new events from March through November. The Summit of Strength is a single-day educational event hosted by Cure SMA. Summits are crafted to provide information for people of all ages and types of SMA, and

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Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

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Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in healthy adult volunteers. SRK-015, a highly specific inhibitor of myostatin activation, was observed to be well tolerated with no apparent safety signals. By demonstrating robust and sustained target engagement in humans, pharmacologic effects on

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Community Spotlight: Kristen Smith

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The SMA community has been lobbying state and federal government for years. Kristen Smith shares how she became involved with advocacy. Kristen Smith describes herself as an artist and advocate. A powerful combination during a time where people across the country are standing up for their community and speaking out for their rights. Kristen is

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