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

    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.

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