229 results on '"West, Michael L."'
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2. Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease
3. Initiation of pharmacologic chaperone therapy for Fabry disease in the Canadian Fabry Disease Initiative (CFDI) registry is not associated with reduction of kidney function
4. Retrospective review of recent ASA prescribing practices for primary prevention of major adverse cardiovascular events in the Canadian Fabry Disease Initiative cohort
5. FollowME Fabry Pathfinders Registry: Renal effectiveness in a cohort of patients on migalastat treatment for at least three years
6. Glycosphingolipid evaluation for Fabry disease patients receiving migalastat after switching from enzyme replacement therapy
7. Chest pain subtype prevalence in the British Columbia cohort of the Canadian Fabry Disease Initiative
8. Facilitating intrafamily communication to enable earlier diagnosis of Fabry disease in relatives: Expert opinion
9. Switch from enzyme replacement therapy to pharmacologic chaperone: Improvement in advanced Fabry nephropathy
10. Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry
11. Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?
12. Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products
13. Lentivirus-mediated gene therapy for Fabry disease
14. Fabry disease biomarkers in patients switched from enzyme replacement therapy to migalastat oral chaperone therapy
15. The spectrum of podocyte injury in later onset (LO) variants of Fabry disease (FD)
16. FollowME Fabry Pathfinders registry: Renal effectiveness in a multi-national, multi-center cohort of patients on migalastat treatment for at least three years
17. Clinical characteristics of female patients enrolled in the FollowME Fabry Pathfinders registry
18. Corrigendum to “Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing” [2021 Sep-Oct;134(1–2):117–131]
19. Long-term outcomes in patients with Fabry disease who were treated with agalsidase alfa for more than nineteen years: The Fabry Outcome Survey
20. Early therapy in Fabry disease: Outcomes from the Canadian Fabry Disease Initiative (CFDI) registry
21. Early initiation of agalsidase alfa treatment improves clinical outcomes in male patients with classical Fabry disease: A Fabry Outcome Survey (FOS) analysis
22. Uncertain Diagnosis of Fabry Disease in Patients with Neuropathic Pain, Angiokeratoma or Cornea Verticillata: Consensus on the Approach to Diagnosis and Follow-Up
23. Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
24. Additional file 1 of Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?
25. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
26. Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing
27. How well does urinary lyso-Gb 3 function as a biomarker in Fabry disease?
28. Gender-specific plasma proteomic biomarkers in patients with Anderson–Fabry disease
29. Canadian Fabry disease registry study group: Report on the A143P Nova Scotia genotype
30. Lyso Gb3 and Gb3 analogues in Fabry disease patients with A143P genotype: A cross-sectional analysis by the CFDR study group
31. Primary Hyperoxaluria Type 1 (PH1) Presenting With End-Stage Kidney Disease and Cutaneous Manifestations in Adulthood: A Case Report
32. Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis
33. Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative
34. Therapeutic challenges in two adolescent male patients with Fabry disease and high antibody titres
35. The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process.
36. Routes to diagnosis of Fabry disease according to patient age and geographic distribution
37. Use of denosumab to treat refractory hypercalcemia in a peritoneal dialysis patient with immobilization and tertiary hyperparathyroidism
38. Renal-Limited Antiglomerular Basement Membrane Disease Related To Alemtuzumab: A Case Report
39. Determination of the crystal structure and substrate specificity of ananain
40. Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis
41. Cardio-renal outcomes with long-term alfa enzyme replacement therapy : a 10-year Fabry outcome survey (FOS) analysis
42. Solid-Phase Oligosaccharide Chemistry and Its Application to Library Synthesis
43. NMR solution structure of the RNA-binding peptide from human immunodeficiency virus (type 1) Rev
44. Targeting HIV-1 protease: a test of drug-design methodologies
45. Patients with Fabry disease on dialysis in the United States
46. FP114RENOPROTECTIVE EFFECT OF AGALSIDASE ALFA IN FABRY DISEASE IS INDEPENDENT OF TYPE OF MUTATION: RESULTS OF 12-YEAR FOLLOW-UP
47. Valvular heart disease complicating advanced Fabry disease: Association with chronic kidney disease
48. FACTs Fabry gene therapy clinical trial: Two-year data
49. Renal and cardiac outcomes in female patients with Fabry disease treated with agalsidase beta: A Fabry registry analysis of pre- versus post-treatment comparison
50. Elevated Inflammatory Plasma Biomarkers in Patients With Fabry Disease: A Critical Link to Heart Failure With Preserved Ejection Fraction
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