European Committee approves world's first gene therapy for children

Discussion in 'Biology & Genetics' started by Plazma Inferno!, Apr 13, 2016.

  1. Plazma Inferno! Ding Ding Ding Ding Administrator

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    Italian scientists along with pharmaceutical company GlaxoSmithKline just developed the world’s first gene therapy, called Strimvelis, available to children who are suffering with ADA Severe Combined Immune Deficiency (ADA-SCID).
    In Europe, approximately 15 children are born with the rare genetic disorder every year. The disease prevents their bodies from creating a specific type of white blood cell. The disease is often called "bubble baby" syndrome, since they must exist in a completely germ-free environment.
    Their immune systems are nearly non-existent, so sufferers rarely live beyond two years. The only known way to cure the disease is with a matching bone marrow donor. Without one, survival rates are extremely low.

    And the European Medicines Agency (EMA) just gave the green light on its testing and production.

    http://futurism.com/european-committee-approves-worlds-first-gene-therapy-children/
     

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