<unk>  is a glucosylceramide synthase inhibitor that is FDA approved for the treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.   Common adverse reactions include diarrhea, weight loss, stomach pain, gas, nausea, vomiting, headache, migraine, tremor, leg cramps, dizziness, weakness, vision problems, thrombocytopenia, muscle cramps, back pain, constipation, dry mouth, heaviness in arms and legs, memory loss, unsteady walking, anorexia, indigestion, paresthesia, stomach bloating, stomach pain not related to food, and menstrual changes.
Miglustat
Migalastat
Eliglustat
Olaflur
Cobicistat
Vintafolide
Mipomersen
Plerixafor