Therapeutic or Preventive Procedure: Enzyme Replacement Therapy

Medical news and research articles

Rare, deadly genetic disease successfully treated in utero for first time

Using a protocol developed at UC San Francisco, physicians have successfully treated a fetus with a devastating genetic disorder for the first time, and the child is now thriving as a toddler, a case study in the New England ...

Nov 10, 2022

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Enzyme therapy shows promise for childhood dementia

Studies into a rare nervous system disorder that causes dementia and early death in children have highlighted a possible treatment for the condition.

Oct 11, 2022

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FDA approves first treatment for acid sphingomyelinase deficiency

The U.S. Food and Drug Administration has approved Xenpozyme (olipudase alfa) for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), a rare genetic disease.

Sep 5, 2022

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