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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 (ISIS) initiated a pivotal phase III study (CHERISH) on ISIS-SMNRx for the treatment of children suffering from spinal muscular atrophy.
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 ...
Fishers High School junior Betsy Swetnam has spinal muscular atrophy.
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
More than 200 supporters of children with spinal muscular atrophy helped raise more than $110,000 at the fourth annual Gala of Hope.
SPINAL muscular atrophy (SMA) is a genetic disorder that affects nerves responsible for muscle function.
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 ...
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, 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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