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Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|May 29, 2007
Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophyDonald Beam, Michele D Poe, James M Provenzale, et al.
Journal of Patient-Reported Outcomes|April 25, 2022
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndromeKatherine Ackerman Porter, Cara O'Neill, Elise Drake, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|November 20, 2020
Toward newborn screening of metachromatic leukodystrophy: results from analysis of over 27,000 newborn dried blood spotsXinying Hong, Jessica Daiker, Martin Sadilek, et al.
Molecular Genetics and Metabolism|April 9, 2021
Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe DiseaseRobert Thompson-Stone, Margie A Ream, Michael Gelb, et al.
Molecular Genetics and Metabolism|January 13, 2019
Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIBChester B Whitley, Suresh Vijay, Bert Yao, et al.
The New England Journal of Medicine|May 20, 2005
Transplantation of umbilical-cord blood in babies with infantile Krabbe's diseaseMaria L Escolar, Michele D Poe, James M Provenzale, et al.
Movement Disorders Clinical Practice|March 7, 2019
A Scale to Assess Activities of Daily Living in Pantothenate Kinase-Associated NeurodegenerationRandall D Marshall, Abigail Collins, Maria L Escolar, et al.
The New England Journal of Medicine|May 7, 2004
Cord-blood transplants from unrelated donors in patients with Hurler's syndromeSusan L Staba, Maria L Escolar, Michele Poe, et al.
Journal of Inherited Metabolic Disease|March 13, 2015
Measurement of psychosine in dried blood spots--a possible improvement to newborn screening programs for Krabbe diseaseColeman T Turgeon, Joseph J Orsini, Karen A Sanders, et al.
Blood Advances|July 8, 2020
Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disordersMark T Vander Lugt, Xiaohua Chen, Maria L Escolar, et al.
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