Author name: Morgan Tuttle

Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study

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Biogen and Ionis Pharmaceuticals announced that SPINRAZA (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function […]

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AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101

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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 announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as a comparator, and enroll approximately 20 patients. This

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Family Registration for the 2017 Annual SMA Conference is Now Open

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Registration is now open for the 2017 Annual SMA Conference! The conference will be held at Disney’s Contemporary Resort at Disney World in Orlando, FL, from Thursday, June 29, through Sunday, July 2, 2017. We are excited to experience Disney together for a second straight year. Disney characters will be at many of our events,

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Spotlight Innovation Launches Development of STL-182 to Treat SMA

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Spotlight Innovation has obtained an exclusive, worldwide license to STL-182, a novel compound invented by Drs. Elliot Androphy at Indiana University and Kevin Hodgetts, director of the Laboratory for Drug Discovery in Neurodegeneration at Brigham and Women’s Hospital. The investigators’ successful research collaboration was supported in part by a series of grants from the National

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6th Annual Congressional Dinner Furthers SMA Advocacy Work

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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 important evening of collaboration. We are excited to have a number of special guests join us, including Rep. Kevin Brady, Rep. Sean Duffy, Rep. Jeb Hensarling, Rep. Bill Huizenga, Rep.

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Cure SMA-funded Researcher Sara Custer Publishes Paper

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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 PLOS ONE . The research in this paper was funded by a basic research grant from Cure SMA. Individuals with SMA don’t produce enough survival motor neuron (SMN) protein, due

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

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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 Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to testify directly to the FDA. The PFDD meeting will include testimony on a

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Cure SMA and Ionis Announce 2016 Holiday Card Contest

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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 with spinal muscular atrophy, their siblings, and to children of parents with SMA. Ionis will select three entries to use in their annual holiday card. These winning entries will receive

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

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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 data from the ongoing Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 as of September 15, 2016. The data were presented by Jerry Mendell, M.D., director

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Biogen Presents New Data at World Muscle Society Congress

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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 late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. The presentations included safety results from the interim analysis of the Phase 3 ENDEAR study in infantile-onset SMA

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