Asfotase alfa
Approved for a growth disorderHeight measured in peopleBy injectionProtein or enzymeAlso Strensiq
The missing enzyme itself, engineered to stick to bone and given by injection to children born without it.
Bone-targeted recombinant human tissue-nonspecific alkaline phosphatase, the enzyme that is missing in hypophosphatasia, given by injection. FDA approved in October 2015, with a label covering perinatal/infantile- and juvenile-onset hypophosphatasia.
Risks. It carries the strongest class of warning for life-threatening allergic reactions, which can happen minutes after a dose or after more than a year of treatment, and it can lay down calcium in the eyes and kidneys.
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Educational content, not medical advice. Nothing here is a recommendation to take or stop taking anything — talk to a doctor about your own situation.