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![]() Date: 2014-12-02 16:03:24Rare diseases Fabry disease Lysosomal storage diseases Renal function Biomarker Enzyme replacement therapy Clinical trial ERT Glycogen storage disease type II Medicine Health Clinical research | Add to Reading List |
![]() | Migalastat and Enzyme Replacement Therapy Have Comparable Effects on Renal Function in Fabry Disease: Phase 3 Study Results Nicholls, K1*, Bichet, DG2, Giugliani, R3, Hughes, D4, Schiffmann, R5, Wilcox, W6, Skuban, N7, RDocID: 1tiPw - View Document |
![]() | The Journal of Rare Disorders CochraneCornerDocID: 19CDW - View Document |
![]() | 7 November 2014 Dear Committee Re: Life Saving Drugs Programme Review Fabry Support Group of Australia SubmissionDocID: W87d - View Document |
![]() | Minimum Recommended Schedule of Assessments for MPS I Patients Initial Assessments Every 6DocID: VV2Y - View Document |
![]() | Pancreatic enzyme replacement therapyDocID: V3Jk - View Document |