158 results on '"Kühl, Jörn-Sven"'
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2. Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy
3. Screening Newborns for Low T Cell Receptor Excision Circles (TRECs) Fails to Detect Immunodeficiency, Centromeric Instability, and Facial Anomalies Syndrome
4. Incidence of subsequent malignancies after total body irradiation-based allogeneic HSCT in children with ALL – long-term follow-up from the prospective ALL-SCT 2003 trial
5. Peroxisomal very long-chain fatty acid transport is targeted by herpesviruses and the antiviral host response
6. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy
7. Total body irradiation as part of conditioning regimens in childhood leukemia—long-term outcome, toxicity, and secondary malignancies
8. Everolimus and mycophenolate mofetil effectively prevent GvHD in children with severe acute kidney injury undergoing allogeneic HSCT
9. Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adrenoleukodystrophy (CALD)
10. Long-term small-fiber neuropathy and pain sensitization in survivors of pediatric acute lymphoblastic leukemia after stem cell transplantation
11. Correction to: Long-term small-fiber neuropathy and pain sensitization in survivors of pediatric acute lymphoblastic leukemia after stem cell transplantation
12. Chapter 17 - Hematopoietic stem cell therapy and ex vivo gene therapy for X-linked adrenoleukodystrophy
13. Allogeneic haematopoietic stem cell transplantation with myeloablative conditioning for adult cerebral X-linked adrenoleukodystrophy
14. Case report: Treatment of advanced CSF1-receptor associated leukoencephalopathy with hematopoietic stem cell transplant
15. The minimum required level of donor chimerism in hereditary hemophagocytic lymphohistiocytosis
16. Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents
17. Effective Immunological Guidance of Genetic Analyses Including Exome Sequencing in Patients Evaluated for Hemophagocytic Lymphohistiocytosis
18. Human CARMIL2 deficiency underlies a broader immunological and clinical phenotype than CD28 deficiency
19. Life‐threatening systemic rotavirus infection after vaccination in severe combined immunodeficiency (SCID)
20. 3 - Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study
21. Incidence of Secondary Malignancies after Total Body Irradiation-Based Allogeneic HSCT in Children with ALL – Long-Term Follow-up from the Prospective ALL-SCT 2003 Trial
22. Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study
23. Correction to: Long-term small-fiber neuropathy and pain sensitization in survivors of pediatric acute lymphoblastic leukemia after stem cell transplantation
24. Efficacy of Brincidofovir in Pediatric Stem Cell Transplant Recipients With Adenovirus Infections
25. Total body irradiation as part of conditioning regimens in childhood leukemia—long-term outcome, toxicity, and secondary malignancies
26. Concurrent axon and myelin destruction differentiates X‐linked adrenoleukodystrophy from multiple sclerosis
27. Disease Stabilization Following Treatment with elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Interim Results from Phase 2/3 (ALD-102) and Phase 3 (ALD-104) Studies (2064)
28. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Cerebral Adrenoleukodystrophy: Effects of Donor Cell Source and Match, Conditioning Regimen, and Stage of Cerebral Disease
29. Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study
30. Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation
31. Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience
32. Risk factors for mixed chimerism in children with hemophagocytic lymphohistiocytosis after reduced toxicity conditioning
33. Vorinostat in the acute neuroinflammatory form of X‐linked adrenoleukodystrophy
34. Lenti-D Hematopoietic Stem Cell Gene Therapy Stabilizes Neurologic Function in Boys with Cerebral Adrenoleukodystrophy
35. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
36. Transplant- and Disease-Related Outcomes of Allogeneic Hematopoietic Stem Cell Transplant in Patients with Cerebral Adrenoleukodystrophy Vary By Donor Cell Source, Conditioning Regimen, and Stage of Cerebral Disease
37. Neurological manifestations of chronic graft-versus-host disease after allogeneic haematopoietic stem cell transplantation: report from the Consensus Conference on Clinical Practice in chronic graft-versus-host disease
38. Effects of the methoxymorpholino derivative of doxorubicin and its bioactivated form versus doxorubicin on human leukemia and lymphoma cell lines and normal bone marrow
39. 92 - Incidence of Secondary Malignancies after Total Body Irradiation-Based Allogeneic HSCT in Children with ALL – Long-Term Follow-up from the Prospective ALL-SCT 2003 Trial
40. Risk factors for mixed chimerism after stem cell transplantation with treosulfan or melphalan based conditioning regimens in children and adolescents with primary hemophagocytic lymphohistocytosis
41. Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents with GATA2-Related Myelodysplastic Syndrome
42. Outcomes of Allogeneic Hematopoietic Stem Cell Transplant in Patients with Cerebral Adrenoleukodystrophy (CALD): Results from an Ongoing, Large, Multicenter, Observational Study
43. Allogeneic hematopoietic stem cell transplantation with myeloablative conditioning for adult cerebral X‐linked adrenoleukodystrophy
44. Outcome of chronic granulomatous disease ‐ Conventional treatment vs stem cell transplantation.
45. Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease—A single center experience.
46. 381 - Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Cerebral Adrenoleukodystrophy: Effects of Donor Cell Source and Match, Conditioning Regimen, and Stage of Cerebral Disease
47. 123 - Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study
48. Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy
49. Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy
50. Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Juvenile Metachromatic Leukodystrophy Compared With Nontransplanted Control Patients
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