March 2019

Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires

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Cure SMA and FAME (Families of SMA, Argentina) have awarded a $200,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, for his project, “Epigenetics in SMN2 E7 Alternative Splicing II.” Epigenetics refers to changes that affect how much protein is produced from each gene, without altering the DNA sequence. Dr. […]

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Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

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Cure SMA has awarded a $200,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, “Neurofilaments as markers of neurodegeneration in SMA.” Dr. Sumner and her team are looking at the neuronal-specific cytoskeletal protein neurofilaments (NFs) released during axonal degeneration. These NFs can be measured in blood and/or cerebral spinal fluid

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The Winter 2019 issue of Compass in Now Available Online

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The Winter 2019 issue of Compass is now available online. This issue covers Cure SMA’s latest drug discovery grant funding and features an updated SMA drug pipeline. Drug discovery, also called translational research, is the second step in the drug development process. Drug discovery takes the seed ideas discovered in basic research, the first stage,

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Team Cure SMA New York City Half Marathon Runners Raise $15,000 for Cure SMA

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Nine Team Cure SMA runners completed the 2019 United Airlines New York City Half Marathon on Sunday, March 17. Together, the runners successfully raised over $15,000 for Cure SMA, the most in the race’s history for Team Cure SMA. The race celebrated its 14th annual run this year and brought more than 20,000 runners onto

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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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