title: Overcoming the Next Barriers to Successful Therapy.
authors.forename: Ian J
authors.initials: IJ
authors.lastname: Cohen
authors.forename: Hagit
authors.initials: H
authors.lastname: Baris
authors.forename: Pramod Kumar
authors.initials: PK
authors.lastname: Mistry
authors.forename: Mark S
authors.initials: MS
authors.lastname: Sands
mesh_terms: D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives / Q000627:therapeutic use; D000284:Administration, Oral; D002482:Cellulose; D018552:Daucus carota; D016503:Drug Delivery Systems; D055808:Drug Discovery; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D056947:Enzyme Replacement Therapy; D000795:Fabry Disease / Q000188:drug therapy; D005776:Gaucher Disease / Q000188:drug therapy; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000188:drug therapy*; D009081:Mucolipidoses / Q000188:drug therapy; D011759:Pyrrolidines / Q000627:therapeutic use; D035583:Rare Diseases / Q000188:drug therapy*
chemical_list: D004791:Enzyme Inhibitors; D011759:Pyrrolidines; D017485:1-Deoxynojirimycin; D002482:Cellulose; C090092:migalastat; C522917:eliglustat