换一批Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study.
第一作者：
Christoph,Wanner
第一单位：
Department of Medicine, Division of Nephrology, University Hospital Würzburg, Oberduerrbacher Str. 6, 97080 Würzburg, Germany.
作者：
关键词
AE, adverse eventBPI-SF, Brief Pain Inventory-Short FormBPI-SF3, Brief Pain Inventory-Short Form item 3BSS, Bristol stool scaleCD, cognitive debriefingCE, concept elicitationCESD-R-20, Center for Epidemiologic Studies Depression Scale RevisedCKD-EPI, Chronic Kidney Disease Epidemiology CollaborationCTCAE, Common Terminology Criteria for Adverse EventsECG, electrocardiographyEOS, end of studyEOT, end-of-treatmentERT, enzyme replacement therapyFABPRO-GI, FABry Disease Patient-Reported Outcome-GastroIntestinalFD, Fabry diseaseFGID, functional gastrointestinal disordersFabry diseaseGCS, glucosylceramide synthaseGI, gastrointestinalGSRS, Gastrointestinal Symptom Rating ScaleGb3, globotriaosylceramideHbA1c, hemoglobin A1cIBS, irritable bowel syndromeIRB, independent review boardLVEF, left ventricular ejection fractionLVMI, left ventricular mass indexLucerastatMODIFYNPSI, neuropathic pain symptom inventoryNRS-11, 11-point numerical rating scaleNYHA, New York Heart AssociationNeP, neuropathic painOLE, open-label extensionPGIC-DS, Patient Global Impression of Change in Disease SeverityPGIC-PS, Patient Global Impression of Change in neuropathic Pain SeverityPGIS-D, Patient Global Impression of Severity of DiseasePGIS-P, Patient Global Impression of Severity of neuropathic painPK, pharmacokineticsPRO, patient-reported outcomeSD, standard deviationSF-36v2, 36-Item Short Form Health Survey Version 2SRT, substrate reduction therapySubstrate reduction therapyUCI, University of California, IrvineUT, usability testingb.i.d., twice dailyeGFR, estimated glomerular filtration rateα-GAL A, lysosomal enzyme α-galactosidase
DOI
10.1016/j.ymgmr.2022.100862
PMID
35782623
发布时间
2022-07-16
- 浏览4
Molecular genetics and metabolism reports
2022年31卷
100862页
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