We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on SMA Pathology. The session was moderated by Mark Rich, MD, PhD SMA Pathology: Consequences and Potential Endpoints This session focused on the circuits that […]
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Thanks to our vibrant community, SMA Awareness Month is off to a fantastic start. Through the Cure SMA Facebook page, and through your own posts, pictures, shares, comments, and outreach, we’ve already reached millions of people since August began. Here are the top five breakthroughs we’ve seen so far. #CureSMA was the #1 trending topic
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We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on drug discovery. The session was moderated by Elliot Androphy, MD Preclinical and Clinical Drug Development for SMA The 2015 SMA Researcher Meeting closed with
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We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on clinical research and outcome measures. The session was moderated by Thomas Crawford, MD. Clinical Research Studies and Outcome Measures This session started off with
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We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on newborn screening. The session was moderated by Kathryn Swoboda, MD. Newborn Screening for SMA: Ethics, Rationale, and Implementation Data generated in mouse models of
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At the Annual SMA Conference, representatives from six of the seven programs currently in clinical trials gave an update on their programs. Of these six programs, four—gene therapy, ISIS-SMNRx, RG7800, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to correct the mutation in the SMN1 gene that causes SMA, and the other three
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We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers the special session, “Emerging Trends in Motor Neuron Pathobiology.” This summary was written by Cure SMA Scientific Advisory Board member Doug Kerr, MD, PhD. Emerging Trends in
2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology Read More »
The House of Representatives has passed 21st Century Cures. Cure SMA is proud to support this legislation. Thank you for your calls and tweets to voice your support to your Representatives. They really made a difference! So what happens next? The Senate is busy drafting their version of the bill, called “Innovation,” and we expect
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Each year, while much of our community is gathered together, we use the Annual SMA Conference to recognize several individuals, families and foundations who have provided special support to the Annual SMA Conference and the SMA community. Here are this year’s awardees. On behalf of the entire community, thank you for all you do! Shaina
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At last week’s Annual SMA Conference, Cytokinetics released additional data from their Phase 1 study of CK-2127107, a skeletal muscle activator. Cytokinetics, in partnership with Astellas, has already announced that a Phase 2 study will begin later this year. Our partnership with Cytokinetics highlights two strengths of Cure SMA’s research model: providing seed funding to
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