618 results on '"Mahlangu, Johnny"'
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2. Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A
3. Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review
4. Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy
5. FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic
6. Emicizumab is efficacious in people with hemophilia A with comorbidities aged ≥50 years: analysis of 4 phase III trials
7. Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies
8. Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks
9. Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1
10. Subcutaneous engineered factor VIIa marzeptacog alfa (activated) in hemophilia with inhibitors: Phase 2 trial of pharmacokinetics, pharmacodynamics, efficacy, and safety
11. Efficacy and safety of subcutaneous prophylaxis with dalcinonacog alfa in adults with haemophilia B
12. Randomized Phase 1b CROSSWALK-a and Phase 2a CROSSWALK-c Trials Crovalimab for SCD
13. Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults
14. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B
15. Reclassifying hemophilia to include the definition of outcomes and phenotype as new targets
16. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study
17. Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis
18. The translational gap for gene therapies in low- and middle-income countries
19. Emicizumab is efficacious in people with hemophilia A with comorbidities aged ≥50 years: analysis of four Phase III trials
20. Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies
21. A randomized, placebo-controlled, double-blind trial of canakinumab in children and young adults with sickle cell anemia
22. Recombinant single‐chain factor VIII in severe hemophilia: Long‐term safety and efficacy in previously treated patients in the AFFINITY extension study
23. Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective
24. Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
25. FRONTIER1: a partially randomized phase 2 study assessing the safety, PK, and PD of Mim8, a factor VIIIa mimetic
26. Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database
27. Application of a hemophilia mortality framework to the Emicizumab Global Safety Database
28. Reduced Volume and Faster Infusion Rate of Activated Prothrombin Complex Concentrate: A Phase 3b/4 Trial in Adults with Hemophilia A with Inhibitors
29. Diagnosis and treatment challenges in lower resource countries: State‐of‐the‐art.
30. Hemophilia gene therapy knowledge and perceptions: Results of an international survey
31. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors
32. Capacity building for inherited bleeding disorders in sub-Saharan Africa
33. Improving access to hemophilia care in sub-Saharan Africa by capacity building
34. Using pharmacokinetics for tailoring prophylaxis in people with hemophilia switching between clotting factor products: A scoping review
35. Phase 3 Trial of Concizumab in Hemophilia with Inhibitors
36. Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List
37. Implementation of a gene therapy education initiative by the ASGCT and Muhimbili University of Health and Allied Sciences
38. RNA sequencing suggests that non‐coding RNAs play a role in the development of acquired haemophilia
39. Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
40. Access to Systemic Anticancer Treatment and Radiotherapy Services in Sub-Saharan Africa
41. Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials
42. Hepcidin and GDF-15 are potential biomarkers of iron deficiency anaemia in chronic kidney disease patients in South Africa
43. Corrigendum to ‘Efficacy of emicizumab is maintained throughout dosing intervals for bleed prophylaxis’ [Research and Practice in Thrombosis and Haemostasis, Volume 7, Issue 2, February 2023, 100077]
44. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study
45. Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List
46. Post hoc longitudinal assessment of efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B
47. Leave no one behind: Optimal Care of all Patients with Haemophilia A.
48. Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A
49. Underdiagnosis of iron deficiency anaemia in HIV-infected individuals: a pilot study using soluble transferrin receptors and intensive bone marrow iron stores to improve the diagnosis.
50. Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)
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