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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 Sanofi’s 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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Discontinuation rates due to adverse events in major trials were relatively low, generally under 10%, but higher than placebo