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FDA Press Release on First Treatment for Menkes Disease Rare Disease: FDA Expands Treatment Approval for Gaucher Disease The FDA expanded the approval of Sanofis Cerezyme (imiglucerase) intravenous infusion enzyme replacement therapy to treat non-neurological symptoms in patients with Type 3 Gaucher disease, which is the rarest and most serious type

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Ozempic contains semaglutide and is FDA-approved for type 2 diabetes, though it is sometimes prescribed off-label for weight loss