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Blood|May 19, 2011
Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort reportWeston P Miller, Steven M Rothman, David Nascene, et al.Neurology|July 4, 2020
Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophyElizabeth I Pierpont, David R Nascene, Ryan Shanley, et al.Parasitology Research|January 7, 2021
Pre-clinical evaluation of the effect of co-medication with antibiotics and oral steroids in Göttingen Minipigs on the biological activity of the probiotic medicinal product TSO (Trichuris suis ova)M V Prosberg, H Kringel, J S Kapel, et al.Molecular Genetics and Metabolism|April 6, 2023
Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type IIMiles C Smith, Lalitha R Belur, Andrea D Karlen, et al.Stem Cells (Dayton, Ohio)|July 29, 2025
Loss of peroxisome function promotes oxidative stress induced hematopoiesisEmma A Schindhelm, Amada Blake, Megan M Constans, et al.Global Mental Health (Cambridge, England)|June 10, 2017
Developing mental health research in sub-Saharan Africa: capacity building in the AFFIRM projectM Schneider, K Sorsdahl, R Mayston, et al.Pediatric Research|August 22, 2019
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantationLynda E Polgreen, Troy C Lund, Elizabeth Braunlin, et al.Scientific Reports|March 21, 2018
Publisher Correction: Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1Elina Makino, Helen Klodnitsky, John Leonard, et al.Scientific Reports|March 1, 2018
Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1Elina Makino, Helen Klodnitsky, John Leonard, et al.Molecular Therapy. Methods & Clinical Development|February 20, 2024
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II miceMiles C Smith, Lalitha R Belur, Andrea D Karlen, et al.Pageof 55