Asfotase alfa in hypophosphatasia in childhood/adolescence: Survival benefit for infants

Hypophosphatasia (HPP) is a rare congenital metabolic disorder. The frequency of severe courses of disease is estimated at 1:100 000: A deficiency of the enzyme phosphatase results in insufficient mineralization of the bones and thus in severe skeletal malformations. The course of disease varies greatly—the earlier the onset of the disease, the more severe are symptoms and complaints. Affected infants in whom the disease occurs before the age of 6 months often die of it and at a very early age, whereas the symptoms are sometimes less pronounced when the disease occurs later.
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