Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa

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Cure SMA and Cure SMA Canada have awarded a $150,000 research grant to Jocelyn Cote, PhD, at the University of Ottawa for his project, “Investigating the contributions of CARM1 and HuR misregulation to SMA skeletal muscle and NMJ defects.” While an essential role for SMN in spinal cord motor neurons remains central to the pathophysiology […]

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What is the Summit of Strength Program?

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Cure SMA is thrilled to continue to provide the Summit of Strength Program to local communities throughout the United States. The 2018 Summit of Strength Program was a great success, and the 2019 Summit of Strength Program has officially begun! Spinal muscular atrophy (SMA) is a disease that robs people of strength by affecting the

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We’re Celebrating Local Support

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We’re doing everything we can to improve quality of life for children, adults and families affected by SMA. We give individuals and families the information and resources they need to live active, engaged, and hopeful lives today. We’re dedicating the month of April to Local Support. The Summit of Strength program is the centerpiece of

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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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