2014 SMA Researcher Meeting Summary: Clinical Trials

We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the last of four total updates, and the second of two in drug discovery and clinical trials. This session was moderated by Douglas Kerr, MD, PhD, Biogen Idec. […]

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2014 SMA Researcher Meeting Summary: Therapy Development

We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the third of four total updates, and the first of two in drug discovery and clinical trials. This summary was written by Cure SMA Scientific Advisory Board member

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Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award

We are proud to announce Scott Geller, who chaired the Chesapeake Chapter Golf Outing for fourteen years, as the first ever recipient of the Annual Community Partnership Award. The award was presented at The Annual SMA Conference this June in Washington D.C. Scott first attended the Chesapeake Chapter Golf Outing as a guest. Scott was

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2014 SMA Researcher Meeting Summary: SMA Pathology

We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the second of four total updates, and the second of two in basic research. This summary was written by Cure SMA Scientific Advisory Board (SAB) member Samuel Pfaff,

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2014 SMA Researcher Meeting Summary: The SMN Protein

We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the first of four total updates, and the first of two in basic research. This summary was written by Cure SMA Scientific Advisory Board member Adrian Krainer PhD,

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Cure SMA Announces New Subcommittee on Adults with SMA

Cure SMA recently announced the formation of the Subcommittee on Adult Outreach. This group will work to actively engage the adult SMA community in order to effectively support them. The population of adults with SMA is increasing, so this new subcommittee will play a vital role in shaping our programs in 2015 and beyond. Initially,

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Isis Releases Phase II Clinical Trial Results

Today, Isis released results from their Phase II open-label clinical trials of ISIS-SMNRx. These results covered two separate trials: a trial in infants with SMA type I, and a trial in children with SMA type II. Isis previously announced recruitment for a Phase III trial of infants with SMA type I. This is the first-ever

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South Florida Gala of Hope Closes in on Milestone

On Thursday, October 2, Jennifer Miller Smith and Fiorenna Fuentes Israel, two dedicated mothers of children with SMA, planned an evening to be remembered in their quest to achieve a huge fundraising milestone: $500,000 to cure spinal muscular atrophy. This year’s event, the 4th Annual Gala of Hope, has already raised $110,000 and climbing, bringing

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