Miller, Weston P.

Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 604

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

Miller, W. P., Rothman, S. M., Nascene, D., Kivisto, T., DeFor, T. E., Ziegler, R. S., Eisengart, J., Leiser, K., Raymond, G., Lund, T. C., et al. (2011) · Blood

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 290

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

Raymond, G. V., Aubourg, P., Paker, A., Escolar, M., Fischer, A., Blanche, S., Baruchel, A., Dalle, J.-H., Michel, G., Prasad, V., et al. (2019) · Biology of Blood and Marrow Transplantation

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 142

A report on state-wide implementation of newborn screening for X-linked Adrenoleukodystrophy

Wiens, K., Berry, S. A., Choi, H., Gaviglio, A., Gupta, A., Hietala, A., Kenney‐Jung, D., Lund, T., Miller, W., Pierpont, E. I., et al. (2019) · American Journal of Medical Genetics Part A

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 75

Neurocognitive trajectory of boys who received a hematopoietic stem cell transplant at an early stage of childhood cerebral adrenoleukodystrophy

Pierpont, E. I., Eisengart, J. B., Shanley, R., Nascene, D., Raymond, G. V., Shapiro, E. G., Ziegler, R. S., Orchard, P. J., & Miller, W. P. (2017) · JAMA Neurology

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 62

Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison’s disease improves survival and neurological outcomes

Polgreen, L. E., Chahla, S., Miller, W., Rothman, S., Tolar, J., Kivisto, T., Nascene, D., Orchard, P. J., & Petryk, A. (2011) · European Journal of Pediatrics

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 49

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

Pierpont, E. I., Nascene, D. R., Shanley, R., Kenney-Jung, D. L., Ziegler, R. S., Miller, W. P., Gupta, A. O., Lund, T. C., Orchard, P. J., & Eisengart, J. B. (2020) · Neurology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 21