PediatricSurvival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation
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open access
Key Words
Cerebral adrenoleukodystrophy
Natural history
Hematopoietic stem cell transplantation
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Financial disclosure: See Acknowledgments on page 547.
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Current address: Department of Pediatrics and Neurology, Penn State Children's Hospital, Hershey, Pennsylvania.
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Current address: Ovid Therapeutics, Inc, New York, New York.
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Current address: Sangamo Therapeutics, Inc, Richmond, California.
© 2018 American Society for Blood and Marrow Transplantation.