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  • 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 ...

  • 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 ...

  • A Sweet Incentive Assists a Woman in Need
    Laguna Beach local Analisa Andrus manages the self-sufficiency program that helps homeless adults rebuild their lives at Friendship Shelter. Now she is in need herself. She suffers from spinal muscular atrophy, a degenerative, genetic neuromuscular disease that currently has no cure. Family and frie... The post A Sweet Incentive Assists a Woman in Need appeared first on Laguna Beach Independent ...

  • Mum Tania Clarence was under 'unbearable pressure' before killing her three disabled children, husband claims
    Gary Clarence spoke out after Tania was detained in hospital over the deaths of Olivia, four, and twins Ben and Max, three, who all had crippling type two spinal muscular atrophy.

  • Bio Blast Pharma Reports Third Quarter Financial Results
    TEL AVIV, Israel -- Bio Blast Pharma Ltd. , a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, today ...

  • Anything for Auni: Community rallies to help child with spinal muscular atrophy
    Auni Naulu was diagnosed earlier this year with SMA, a genetic disease, but this diagnosis has not stopped her family from seeing the good in the world.

  • Lindale community rallying to give local dad a lift
    Duane Hale was diagnosed with Spinal Muscular Atropy at four years old. He's been in a power chair since the 8th grade.

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