November 2016

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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