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6th Annual Congressional Dinner Furthers SMA Advocacy Work

October 27, 2016
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This year’s 6th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 29, bringing together families, government, and industry for an […]

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Cure SMA-funded Researcher Sara Custer Publishes Paper

October 25, 2016
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Sara Custer, PhD, and her colleagues at Indiana University have published a new paper, “Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells” in the journal […]

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SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA

October 19, 2016
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Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient […]

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Cure SMA and Ionis Announce 2016 Holiday Card Contest

October 13, 2016
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Back by popular demand, Cure SMA and Ionis Pharmaceuticals are once again teaming up for a holiday card contest! This contest is open to children […]

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AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress

October 8, 2016
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AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an update on interim […]

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Biogen Presents New Data at World Muscle Society Congress

October 8, 2016
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New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen and Ionis Pharmaceuticals in the […]

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Cure SMA Announces Newborn Screening Initiative in Partnership with MDA

September 27, 2016
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Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening […]

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Biogen and Ionis Release Community Statement on NDA Filing Completion

September 26, 2016
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Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the the completion of their NDA filing for nusinersen. Dear members of the SMA […]

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Roche Launches New Phase 2 Clinical Trials

September 23, 2016
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Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, […]

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Catch Up on Research News from the 2016 Family Friendly Research Poster Session

September 19, 2016
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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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