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doi: 10.3390/ph17040497 95 GaoQLiGZuYXuYWangCXiangDet al

The mechanisms of this transporting process are still unknown, but it is certain that carnitine acyl-CoA transferase is the key enzyme in this process

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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Gastrointestinal side effects are most common during the first 48 weeks but usually diminish with time