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  • Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Children With Spinal Muscular Atrophy
    CARLSBAD, Calif., Nov. 25, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (ISIS) announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA).  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness.  The Phase 3 study, CHERISH, is the second Phase 3 ...

  • Isis Pharmaceuticals Earns Milestone on Study Initiation
    Isis Pharmaceuticals (ISIS) initiated a pivotal phase III study (CHERISH) on ISIS-SMNRx for the treatment of children suffering from spinal muscular atrophy.

  • Spinal Muscular Atrophy Program Enters Phase 1b/2a Trial in Patients with SMA
    SOUTH PLAINFIELD, N.J., Nov. 19, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation ...

  • Shot of wheelchair actress is prize for IndyStar photographer
    Fishers High School junior Betsy Swetnam has spinal muscular atrophy.                

  • Isis Pharmaceuticals Earns Milestone on Study Initiation - Analyst Blog
    Isis Pharmaceuticals, Inc. ( ISIS ) has commenced a pivotal phase III study on ISIS SMNRx for the treatment of children suffering from spinal muscular atrophy (SMA). With the initiation of this study, Isis Pharma earned a milestone payment

  • Combating spinal muscular atrophy
    More than 200 supporters of children with spinal muscular atrophy helped raise more than $110,000 at the fourth annual Gala of Hope.

  • Factfile Genetic disorder
    SPINAL muscular atrophy (SMA) is a genetic disorder that affects nerves responsible for muscle function.

  • CSF-Delivered Gene Therapy Shows Promise for Spinal Muscular Atrophy
    ELK GROVE VILLAGE, Ill., Nov. 5, 2014 /PRNewswire-USNewswire/ -- A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Children's Hospital was recently published in the journal Molecular Therapy. The paper, "Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA—a dose response study in mice and nonhuman primates" is the first publication resulting from a ...

  • Local man with MD launches program to help others
    A Bethlehem man diagnosed with Spinal Muscular Atrophy is launching a new program to help others. Shane Burcaw is the founder of the non-profit "Laughing at My Nightmare." Burcaw was diagnosed with the condition shortly after birth and has been in a wheelchair since he was two years old. Today at Moravian College, Burcaw announced a donation of $14,000 to the Muscular Dystrophy Association ...

  • Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA
    Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA).

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