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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. Among authors: miller wp. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation.
Raymond GV, Aubourg P, Paker A, Escolar M, Fischer A, Blanche S, Baruchel A, Dalle JH, Michel G, Prasad V, Miller W, Paadre S, Balser J, Kurtzberg J, Nascene DR, Orchard PJ, Lund T. Raymond GV, et al. Biol Blood Marrow Transplant. 2019 Mar;25(3):538-548. doi: 10.1016/j.bbmt.2018.09.036. Epub 2018 Oct 4. Biol Blood Marrow Transplant. 2019. PMID: 30292747 Free article.
Intensity of MRI Gadolinium Enhancement in Cerebral Adrenoleukodystrophy: A Biomarker for Inflammation and Predictor of Outcome following Transplantation in Higher Risk Patients.
Miller WP, Mantovani LF, Muzic J, Rykken JB, Gawande RS, Lund TC, Shanley RM, Raymond GV, Orchard PJ, Nascene DR. Miller WP, et al. AJNR Am J Neuroradiol. 2016 Feb;37(2):367-72. doi: 10.3174/ajnr.A4500. Epub 2015 Oct 1. AJNR Am J Neuroradiol. 2016. PMID: 26427835 Free PMC article.
130 results