Research

2014 SMA Researcher Meeting Summary: Therapy Development

October 24, 2014
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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. […]

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2014 SMA Researcher Meeting Summary: SMA Pathology

October 22, 2014
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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. […]

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2014 SMA Researcher Meeting Summary: The SMN Protein

October 17, 2014
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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. […]

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Isis Releases Phase II Clinical Trial Results

October 10, 2014
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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 […]

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AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy

October 8, 2014
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AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world’s first human gene therapy […]

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Isis Presents Phase II Trial Results

October 7, 2014
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This Friday, October 10, Isis Pharmaceuticals will be presenting results from their Phase II clinical trials of ISIS-SMNRx in both children and infants. The results […]

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AveXis Receives Orphan Drug Designation

October 6, 2014
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AveXis has received Orphan Drug Designation from the FDA for their gene therapy program, called chariSMA. Orphan Drug Designation is granted by the FDA to […]

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Isis Begins Recruiting for Phase III Trial

October 3, 2014
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Isis is currently recruiting participants for Endear, a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The goal of this […]

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Cure SMA-Funded CALIBR Research Collaboration Expanded to USC

October 1, 2014
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Cure SMA is pleased to announce that we have expanded our research collaboration with the California Institute for Biomedical Research (CALIBR) to include a collaborative […]

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Catch Up on Research News from our Family Friendly Poster Session

September 25, 2014
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Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in […]

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