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Keyword: spinalmuscular

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  • FDA approves $2M medicine, most expensive ever

    05/25/2019 5:24:36 AM PDT · by SMGFan · 59 replies
    NYPost ^ | May 25, 2019
    US regulators have approved the most expensive medicine ever, for a disorder that destroys a baby’s muscle control and kills nearly all of those with the most common variant of the disease within a couple years. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage. The gene therapy, developed by Novartis, treats a rare condition called spinal muscular atrophy, which strikes about 400 babies born in the US each year. The Food and Drug Administration on Friday approved the drug, called Zolgensma, for all children under age 2 who are confirmed by...
  • Powerful video shows that every life matters

    12/23/2014 7:22:09 PM PST · by Morgana · 3 replies
    Live Action News ^ | Dec 23, 2014 | Cassy Fiano
    Many people don’t get the opportunity to come face-to-face with a person who has a disability. Rarer still is getting an honest view of how a person feels about their disability. But millions caught a glimpse into the life of an extraordinary individual living with a disability after actor Ashton Kutcher shared the following video with 18 million of his followers on social media. Cashel Gardner is a 17-year-old living with Spinal Muscular Atrophy, which, for Cashel, means that he cannot move, breathe, talk, or eat on his own. But he made this video to show that his life is...