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1-a]isoindol-5(9bH)-one
improvement of bone mineral density
Smiles: C[C@H](NC(=O)[C@@H](O)C1=CC(F)=CC(F)=C1)C(=O)N[C@@H]1C(=O)N(C)C2=CC=CC=C2C2=CC=CC=C21
PubMed PMID: 26354274
Miglustat Hydrochloride 240551 1-a]isoindol-5(9bH)-oneMiglustat, also known as OGT 918 and NB DNJ, is a drug developed by Actelion and is used primarily to treat Type I Gaucher disease (GD1). Miglustat inhibits glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids (it forms glucosylceramide and accumulates within the macrophages). Miglustat is used to treat adults with mild to moderate Type I Gaucher disease and it is the first treatment to be approved for patients
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