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1 . Academic Journal
Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy
저자
by
Chiesa, Robert
;
Boelens, Jaap Jan
;
Duncan, Christine N.
;
Kühl, Jörn-Sven
;
Sevin, Caroline
, et al.
소스
In
Blood Advances
8 March 2022 6(5):1512-1524
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7 . Academic Journal
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)
저자
by
Duncan, Christine N.
;
Orchard, Paul J.
;
Eichler, Florian S.
;
Kühl, Jörn-Sven
;
De Oliveira, Satiro N.
, et al.
소스
In
Transplantation and Cellular Therapy
February 2024 30(2) Supplement:S232-S233
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7 . Academic Journal
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
저자
by
Duncan, Christine N.
;
Kühl, Jörn-Sven
;
Chiesa, Robert
;
Boelens, Jaap-Jan
;
Eichler, Florian
, et al.
소스
In
Transplantation and Cellular Therapy
March 2022 28(3) Supplement:S2-S4
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7 . Academic Journal
Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study.
저자
by
Muenzer, Joseph
;
Burton, Barbara K.
;
Amartino, Hernan M.
;
Harmatz, Paul R.
;
Gutiérrez-Solana, Luis González
, et al.
소스
Orphanet Journal of Rare Diseases
. 11/16/2023, Vol. 18 Issue 1, p1-19. 19p.
Open Access (BioMed Central)
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7 . Academic Journal
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
저자
by
Boelens, Jaap Jan
;
Chiesa, Robert
;
Duncan, Christine N.
;
Kühl, Jörn-Sven
;
Sevin, Caroline
, et al.
소스
In
Transplantation and Cellular Therapy
March 2021 27(3) Supplement:S318-S319
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7 . Academic Journal
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
저자
by
Orchard, Paul J.
;
Duncan, Christine N.
;
Eichler, Florian S.
;
De Oliveira, Satiro N.
;
Thrasher, Adrian J.
, et al.
소스
In
Transplantation and Cellular Therapy
March 2021 27(3) Supplement:S114-S114
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7 . Periodical
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
저자
by
Duncan, Christine N.
;
Kühl, Jörn-Sven
;
Chiesa, Robert
;
Boelens, Jaap-Jan
;
Eichler, Florian
, et al.
소스
Transplantation and Cellular Therapy; March 2022, Vol. 28 Issue: 3, Number 3 Supplement 1 pS2-S4, 3p
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7 . Academic Journal
Timing of diagnosis of patients with pompe disease: Data from the pompe registry
저자
by
Kishnani, Priya S.
;
Amartino, Hernán M.
;
Lindberg, Christopher
;
Miller, Timothy M.
;
Wilson, Amanda
, et al.
소스
American Journal Of Medical Genetics - A
. Oct 01, 2013 161(10):2431-2443
Web of Science
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7 . Academic Journal
Methods of diagnosis of patients with Pompe disease: Data from the Pompe Registry.
저자
by
Kishnani, Priya S.
;
Amartino, Hernán M.
;
Lindberg, Christopher
;
Miller, Timothy M.
;
Wilson, Amanda
, et al.
소스
Molecular Genetics & Metabolism
. Sep2014, Vol. 113 Issue 1/2, p84-91. 8p.
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7 . Academic Journal
Pompe disease (glycogen storage disease type II) in Argentineans: Clinical manifestations and identification of 9 novel mutations
저자
by
Palmer, Rachel E.
;
Amartino, Hernan M.
;
Niizawa, Gabriela
;
Blanco, Mariana
;
Pomponio, Robert J.
, et al.
소스
Neuromuscular Disorders
. Jan2007, Vol. 17 Issue 1, p16-22. 7p.
Full Text (Clinical Key)
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