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Pharmaceutics|November 26, 2022
Prospective Validation and Refinement of a Population Pharmacokinetic Model of Fludarabine in Children and Young Adults Undergoing Hematopoietic Cell TransplantationJordan T Brooks, Belen P Solans, Ying Lu, et al.
Cells|March 10, 2022
Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral AdrenoleukodystrophyHongge Wang, Matthew D Davison, Martin L Kramer, et al.
The New England Journal of Medicine|October 9, 2024
Hematologic Cancer after Gene Therapy for Cerebral AdrenoleukodystrophyChristine N Duncan, Jacob R Bledsoe, Bartosz Grzywacz, et al.
The New England Journal of Medicine|April 3, 2026
Base Editing of HBG1 and HBG2 Promoters for Sickle Cell DiseaseAshish O Gupta, Akshay Sharma, Haydar Frangoul, et al.
The New England Journal of Medicine|October 9, 2024
Lentiviral Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, Christine N Duncan, Patricia L Musolino, et al.
Molecular Genetics and Metabolism|July 4, 2024
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophyLaura Ann Adang, Samuel Groeschel, Chloe Grzyb, et al.
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