Weston P. Miller

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Florian Eichler, Christine Duncan, Patricia L. Musolino et al. · New England Journal of Medicine · 2017 · 595 citations

Medicine, Hematology, Neurology +16

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

Weston P. Miller, Steven M. Rothman, David Nascene et al. · Blood · 2011 · 290 citations

Medicine, Hematology, Pediatrics +8

Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

Alexander A. Boucher, Weston P. Miller, Ryan Shanley et al. · Orphanet Journal of Rare Diseases · 2015 · 183 citations · Full text

Medicine, Hematology, Neurology +15

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

Gerald V. Raymond, Patrick Aubourg, Asif Paker et al. · Biology of Blood and Marrow Transplantation · 2018 · 147 citations · Full text

Medicine, Neurology, Pediatrics +17

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

Perry B. Shieh, Nancy L. Kuntz, Wolfgang Müller‐Felber et al. · The Lancet Neurology · 2023 · 120 citations · Full text

Medicine, Molecular Medicine, Pharmacotherapy +15