26 results on '"Geberhiwot, Tarekegn"'
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2. High sensitivity troponin T and I to evaluate Fabry disease cardiomyopathy
3. Retrospective assessment of family screening in Fabry disease
4. Barriers and facilitators to physical activity and exercise in late-onset Pompe disease (LOPD): A qualitative study exploring patients' perspectives and experiences
5. Potential increased risk of renal cell carcinoma in Fabry disease
6. Investigating the role of miglustat in the management of a patient with Tangier disease: An n-of-1 study with alternating periods of intervention and control
7. Baseline demographics of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in enzyme replacement therapy-experienced adults with late-onset Pompe disease
8. At home enzyme replacement therapy for patients with lysosomal disorders: A single-centre experience
9. Cipaglucosidase alfa and miglustat under the Early Access to Medicines Scheme (EAMS): A single centre experience
10. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study
11. Phenom: a prospective clinical study on the clinical impact of phenylketonuria in adults (307–902)
12. Evaluation of algorithms to identify patients with Fabry disease using routinely collected hospital activity data
13. Proposed stages of cardiomyopathy in Fabry disease
14. Clinical utility of total concentration of globotriaosylsphingosine (Lyso-Gb) and its analogues in diagnosing Fabry disease
15. Long-term clinical outcomes of patients with Morquio syndrome type A treated with elosulfase alfa: Results from a managed access agreement in England
16. Audit on cardiovascular risk in Fabry disease
17. The natural progression of cardiac involvement in Fabry disease
18. Impact of two-year elosulfase alfa treatment on patient-reported outcomes in patients with Morquio syndrome type A: Results from an English managed access agreement
19. A multicentre study of cardiac device implantation, arrhythmic burden and risk factors in Fabry cardiomyopathy
20. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment
21. Safety, efficacy, and immunogenicity of elosulfase alfa in patients with Morquio syndrome type A participating in 2 sequential open-label studies (MOR-002/MOR-100), representing 5 years of treatment
22. Impact of elosulfase alfa treatment on patient-reported outcomes in Morquio syndrome type A: Results from the first year of a managed access agreement in England
23. The subclinical phenotype of cardiac Fabry disease
24. Clinical outcomes in Morquio syndrome type A treated with elosulfase alfa: results from the managed access agreement in England
25. Impact of elosulfase alfa in patients with Morquio syndrome type A who have limited ambulation: An open-label, phase 2 study
26. POM-001 phase 1/2 study of BMN 701, GILT-tagged recombinant human (rh) GAA in late-onset Pompe disease: Initial experience in 22 patients
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