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FDA approves first treatment for a rare genetic disorder – News-Medical.Net
Today, the U.S. Food and Drug Administration approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder.
Reviewed by Emily Henderson, B.Sc.Nov 23 2020
Today, the U.S. Food and Drug Administration approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.
The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input…
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