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Guidance programme

Showing 1 to 2 of 2 results for strimvelis

  1. Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)

    Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency.

  2. Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in babies, children, young people and adults.