608 results on '"Pierce, Glenn F."'
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2. Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy
3. Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
4. The underevaluated impacts of the therapeutic revolution of hemophilia on women and girls
5. The management of liver disease in people with congenital bleeding disorders: guidance from European Association for Haemophilia and Allied Disorders, European Haemophilia Consortium, ISTH, and World Federation of Hemophilia
6. The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients
7. Anti-FVIII antibodies in Black and White hemophilia A subjects: do F8 haplotypes play a role?
8. Hemophilia gene therapy: first, do no harm
9. Bispecific antibodies mimicking factor VIII in hemophilia A: converting innovation to an essential medicine
10. Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years
11. Results of genetic analysis of 11 341 participants enrolled in the My Life, Our Future hemophilia genotyping initiative in the United States
12. Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
13. Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A.
14. Long‐term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post‐treatment
15. Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database
16. Application of a hemophilia mortality framework to the Emicizumab Global Safety Database
17. Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy
18. Deciphering conundrums of AAV liver-directed gene therapy: focus on hemophilia
19. The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients
20. Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List
21. A foundation to better care: Target product profile for haemophilia and Von Willebrand disease point‐of‐care diagnostics in low‐resource settings
22. Adeno-associated virus and hepatocytes: frenemies?
23. Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List
24. Endogenous factor VIII synthesis from the intron 22-inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A.
25. Reported prevalence of von Willebrand disease worldwide in relation to income classification
26. Von Willebrand disease: Gaining a global perspective
27. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study
28. Landmark endorsement of a global registry: The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard.
29. Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding
30. Two gene therapies for hemophilia available: Now what?
31. Development of a target product profile (TPP) for haemophilia point‐of‐care (POC) diagnostic devices for low‐resource countries and remote settings
32. Manufacturing process used to produce long-acting recombinant factor VIII Fc fusion protein
33. Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis
34. First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy
35. Towards achieving a haemophilia-free mind
36. Theory of change and strategic priorities of the world federation of haemophilia
37. Chapter 134 - Hemophilia A and B
38. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
39. Roles of Keratinocyte Growth Factor in Epithelial Growth and Regeneration
40. AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
41. Supporting patients with haemophilia in a world of crises: New role for the WFH and its partners
42. Sixth Åland Island Conference on von Willebrand disease
43. Supporting patients with haemophilia in a world of crises: New role for the WFH and its partners.
44. Evaluation of recombinant factor VIII Fc (Eloctate) activity by thromboelastometry in a multicenter phase 3 clinical trial and correlation with bleeding phenotype
45. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs
46. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
47. Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
48. Eliminating Panglossian thinking in development of AAV therapeutics
49. Theory of change and strategic priorities of the world federation of haemophilia.
50. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment
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