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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.Acta Neuropathologica|June 27, 2023
SMN regulates GEMIN5 expression and acts as a modifier of GEMIN5-mediated neurodegenerationTyler R Fortuna, Sukhleen Kour, Anuradha Venkatakrishnan Chimata, 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.International Journal of Molecular Sciences|August 26, 2022
β-Galactosylceramidase Deficiency Causes Upregulation of Long Pentraxin-3 in the Central Nervous System of Krabbe Patients and <i>Twitcher</i> MiceDaniela Coltrini, Adwaid Manu Krishna Chandran, Mirella Belleri, et al.Blood|July 1, 2008
Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomesVinod K Prasad, Adam Mendizabal, Suhag H Parikh, et al.Pageof 9