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  • Plant City mom on mission to eradicate muscular disease
    PLANT CITY — Shawn Santos has a simple explanation for her mission to raise awareness of spinal muscular atrophy, a rare genetic disease. “We need to find a cure for our babies,” she said.

  • Spinal Muscular Atrophy (SMA) Global Clinical Trials Review, H2, 2014
    NEW YORK, Aug. 11, 2014 /PRNewswire/ -- announces that a new market research report is available in its catalogue: Spinal Muscular Atrophy (SMA) Global Clinical Trials Review, H2, 2014 ...

  • New treatments provide hope for children with Spinal Muscular Atrophy
    Dr. Lisa Baumbach-Reardon, a neurogenomics investigator at Phoenix’s Translational Genomics Research Institute, or TGen, believes there is hope.                

  • Isis Pharmaceuticals Earns $2M from the Advancement of ISIS-SMN Rx in Children with Spinal Muscular Atrophy
    CARLSBAD, Calif., Aug. 15, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (ISIS) announced today that it has earned a $2 million milestone payment from Biogen Idec related to the advancement of the ongoing open-label extension study of ISIS-SMNRx in children with spinal muscular atrophy (SMA).  ISIS-SMNRx is designed to alter the splicing of a closely related gene (SMN2) to increase production ...

  • Plant City mom works tirelessly to help find a cure for daughterand#x2019;s disease
    Shawn Santos has a simple explanation for her mission to raise awareness of spinal muscular atrophy, a rare genetic disease.

  • Woman enjoys magic of restored freedom
    Published: Friday, August 29, 2014 at 12:28 PM. Jessica Hayward’s summer has been one of emotional extremes. The 27-year-old with spinal muscular atrophy went from mobile to bedbound and then back to mobile thanks to the kindness of strangers.

  • Orally Delivered Compounds, Which Selectively Modify RNA Splicing Prevent Deficits In Mouse Models Of Spinal Muscular ...
    SOUTH PLAINFIELD, N.J., Aug. 7, 2014 /PRNewswire/ -- Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in mouse models of Spinal Muscular Atrophy (SMA). Scientists from Roche Pharma Research and Early Development (pRED), PTC Therapeutics ...

  • Left without a taxi ride
    EAST Devonport woman Katrina Rose was born with spinal muscular atrophy type 2 - a disorder caused by a loss of specialised nerve cells in the spinal cord and the part of the brain that is connected to the spinal cord.

  • Weekend TV watch
    North Reading teenager Kate Pecora will be featured on the 49th annual “MDA Show of Strength” (Sunday at 9 p.m. on WCVB, Ch. 5). Pecora lives with spinal muscular atrophy Type II, which leads to muscle weakness, and is participating in a clinical trial. Celebrities appearing on the perennial Labor Day fundraiser include Alyssa Milano, Susan Lucci, Rascal Flatts, LeAnn Rimes and Fall Out Boy.

  • Max's Moment: North Charleston boy battling deadly disease, named firefighter for a day
    Max High is a fighter.  For most of his life, the North Charleston boy has been battling Spinal Muscular Atrophy, or SMA,a disease that severely weakens the muscles, taking away the ability to walk, eat or breathe.

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