34 results on '"Cassiman, David"'
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2. Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study
3. Estimating the broader fiscal consequences of acute hepatic porphyria (AHP) with recurrent attacks in Belgium using a public economic analytic framework
4. D-galactose supplementation in individuals with PMM2-CDG: results of a multicenter, open label, prospective pilot clinical trial
5. Amino acid levels determine metabolism and CYP450 function of hepatocytes and hepatoma cell lines
6. Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients
7. Translatome analysis reveals altered serine and glycine metabolism in T-cell acute lymphoblastic leukemia cells
8. Correction: The ribosomal RPL10 R98S mutation drives IRES-dependent BCL-2 translation in T-ALL
9. Evidence for an alternative fatty acid desaturation pathway increasing cancer plasticity
10. The ribosomal RPL10 R98S mutation drives IRES-dependent BCL-2 translation in T-ALL
11. Liver transplantation for very severe hepatopulmonary syndrome due to vitamin A-induced chronic liver disease in a patient with Shwachman-Diamond syndrome
12. Frequency and pathogenesis of central liver nodules in Alagille syndrome patients
13. Dietary intervention, but not losartan, completely reverses non-alcoholic steatohepatitis in obese and insulin resistant mice
14. Erratum: Corrigendum: Mutations in SNORD118 cause the cerebral microangiopathy leukoencephalopathy with calcifications and cysts
15. Off-label use of orphan medicinal products: a Belgian qualitative study
16. Usefulness of the single-operator cholangioscopy system SpyGlass in biliary disease: a single-center prospective cohort study and aggregated review
17. Mutations in SNORD118 cause the cerebral microangiopathy leukoencephalopathy with calcifications and cysts
18. Neurocognitive outcome in tyrosinemia type 1 patients compared to healthy controls
19. The orphan drug pipeline in Europe
20. Management dilemmas in pediatric nephrology: Cystinosis
21. Reimbursement of orphan drugs in Belgium: what (else) matters?
22. Shining a light in the black box of orphan drug pricing
23. Endoscopic resection of ampullary lesions: a single-center 8-year retrospective cohort study of 91 patients with long-term follow-up
24. Misdiagnosis as asphyxiating thoracic dystrophy and CMV-associated haemophagocytic lymphohistiocytosis in Shwachman-Diamond syndrome
25. Development and validation of COMPASS: clinical evidence of orphan medicinal products – an assessment tool
26. Clinical evidence for orphan medicinal products-a cause for concern?
27. Cost-Effectiveness Assessment of Orphan Drugs
28. PS18 - 87. A novel FPLD-associated PPARgamma mutant (E379K) displays a selective defect in target gene transcription
29. Orphan Drugs for Rare Diseases
30. Are some orphan drugs for rare diseases too expensive? A study of purchase versus compounding costs
31. Drugs for Rare Diseases: Influence of Orphan Designation Status on Price
32. A novel mutation causing mild, atypical fumarylacetoacetase deficiency (Tyrosinemia type I): a case report
33. Hepatitis B virus replication causes oxidative stress in HepAD38 liver cells
34. Expression of neural cell adhesion molecule in human liver development and in congenital and acquired liver diseases
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