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NDRD Publications

 

Clinical management of Krabbe disease. Journal of Neuroscience Research
Escolar ML, West T, Dallavecchia A, Poe MD, LaPoint K. . Journal of Neuroscience Research.2016 Nov 1;94(11):1118-25
https://www.ncbi.nlm.nih.gov/pubmed/?term=27638597

Thickening of the Cauda Equina Roots: a Common Finding in Krabbe Disease
Zuccoli G, Narayanan S, Panigrahy A, Poe MD, Escolar ML. Eur Radiol. 2016 May 2:1-6
https://www.ncbi.nlm.nih.gov/pubmed/?term=27137647

Midbrain morphology reflects extent of brain damage in Krabbe disease
Zuccoli G, Narayanan S, Panigrahy A, Poe MD, Escolar ML. Neuroradiology, 2015 Jul;57(7):739-45
https://www.ncbi.nlm.nih.gov/pubmed/?term=25859833

Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease
Gupta A, Poe MD, Styner MA, Panigrahy a, Escolar ML. Neuroimage Clin. 2014 Sep 26;7:792-8
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4375637/

Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation.
Coletti HY, Aldenhoven M, Yelin K, Poe MD, Kurtzberg J, Escolar ML. JIMD reports. 2015 Jan 23;20:77-86
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4375128/

Early treatment is associated with improved cognition in hurler syndrome
Poe MD, Chagnon SL, Escolar ML. Annals of Neurology. 2014 Nov;76(5):747-53
http://onlinelibrary.wiley.com/doi/10.1002/ana.24246/abstract

Escolar ML, Natural history of Sanfilippo syndrome type A.
Buhrman D, Thakkar K, Poe MD. Journal of Inherited Metabolic Disease. Published online November 2013
http://www.ncbi.nlm.nih.gov/pubmed/?term=escolar+buhrman

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy.
Poe MD, Martin HR, Provenzale JM, Kurtzberg J, Mendizabal A, Escolar ML.Biol Blood Marrow Transplant 2013 Apr;19(4):616-24.
http://www.ncbi.nlm.nih.gov/pubmed/23348427

Correlation of automated volumetric analysis of brain MR imaging with cognitive impairment in a natural history study of mucopolysaccharidosis II.
Fan, Z., Styner, M., Muenzer, J., Poe, M., & Escolar, M. (2010). AJNR Am J Neuroradiol, 31(7), 1319-1323.
http://www.ajnr.org/content/31/7/1319.full

Early clinical markers of central nervous system involvement in mucopolysaccharidosis type II. Holt, J., Poe, M. D., & Escolar, M. L. (2011). J Pediatr, 159(2), 320-326.e2.
http://www.sciencedirect.com/science/article/pii/S0022347611002757

Natural progression of neurological disease in mucopolysaccharidosis type II.
Holt, J. B., Poe, M. D., & Escolar, M. L. (2011). Pediatrics, 127(5), e1258-1265.
http://www.pediatricsdigest.mobi/content/127/5/e1258.full

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients. Mynarek, M., Tolar, J., Albert, M. H., Escolar, M. L., Boelens, J. J., Cowan, M. J., et al. (2012) Bone Marrow Transplant. 47(3):352-359.
http://www.nature.com/bmt/journal/v47/n3/full/bmt201199a.html

DTI registration in atlas based fiber analysis of infantile Krabbe disease.
Wang, Y., Gupta, A., Liu, Z., Zhang, H., Escolar, M. L., Gilmore, J. H., et al. (2011). Neuroimage, 55(4), 1577-1586.
http://www.ncbi.nlm.nih.gov/pmc/articles/pmc3062693/

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Boelens, J. J., Rocha, V., Aldenhoven, M., Wynn, R., O'Meara, A., Michel, G., et al. (2009). Biol Blood Marrow Transplant, 15(5), 618-625.
http://download.journals.elsevierhealth.com/pdfs/journals/1083-8791/PIIS108387910900072X.pdf

Metachromatic leukodystrophy: a case of triplets with the late infantile variant and a systematic review of the literature. Mahmood, A., Berry, J., Wenger, D. A., Escolar, M., Sobeih, M., Raymond, G., et al. (2010). J Child Neurol. 25(5), 572-580.
http://jcn.sagepub.com/content/25/5/572.short

Multidisciplinary management of Hunter syndrome. Muenzer, J., Beck, M., Eng, C. M., Escolar, M. L., Giugliani, R., Guffon, N. H., et al. (2009). Pediatrics, 124(6), e1228-1239.
http://pediatrics.aappublications.org/content/124/6/e1228.long

Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease. Escolar, M. L., Poe, M. D., Smith, J. K., Gilmore, J. H., Kurtzberg, J., Lin, W., et al. (2009). AJNR Am J Neuroradiol, 30(5), 1017-1021.
http://www.ajnr.org/content/30/5/1017.long

Correlation of neurodevelopmental features and MRI findings in infantile Krabbe's disease. Provenzale, J. M., Peddi, S., Kurtzberg, J., Poe, M. D., Mukundan, S., & Escolar, M. (2009). AJR Am J Roentgenol, 192(1), 59-65.
http://www.ajronline.org/doi/full/10.2214/AJR.07.3885

Newborn screening for Krabbe disease: the New York State model. Duffner, P. K., Caggana, M., Orsini, J. J., Wenger, D. A., Patterson, M. C., Crosley, C. J., et al. (2009). Pediatr Neurol, 40(4), 245-252; discussion 253-255.
http://www.pedneur.com/article/S0887-8994(08)00618-8/abstract

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 outcomes. Prasad, V. K., Mendizabal, A., Parikh, S. H., Szabolcs, P., Driscoll, T. A., Page, K., et al. (2008). Blood, 112(7), 2979-2989.
http://bloodjournal.hematologylibrary.org/content/112/7/2979.long

Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective. Martin, H. R., Poe, M. D., Reinhartsen, D., Pretzel, R. E., Roush, J., Rosenberg, A., et al. (2008). Acta Paediatr Suppl, 97(457), 69-75.
http://onlinelibrary.wiley.com/doi/10.1111/j.1651-2227.2008.00651.x/abstract?deniedAccessCustomisedMessage=&userIsAuthenticated=false

Gross motor development of children with hurler syndrome after umbilical cord blood transplantation. Dusing, S. C., Thorpe, D. E., Poe, M. D., Rosenberg, A. E., Mercer, V. S., & Escolar, M. L. (2007 Phys Ther, 87(11), 1433-1440.
http://ptjournal.apta.org/content/87/11/1433.long

Temporal and Spatial Gait Characteristics of Children With Hurler Syndrome After Umbilical Cord Blood Transplantation. Dusing, S. C., Thorpe, D. E., Mercer, V. S., Rosenberg, A. E., Poe, M. D., & Escolar, M. L. (2007). Phys Ther. 87(8), 978-985.
http://ptjournal.apta.org/content/87/8/978.long

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy. Beam, D., Poe, M. D., Provenzale, J. M., Szabolcs, P., Martin, P. L., Prasad, V., et al. (2007). Biol Blood Marrow Transplant, 13(6), 665-674.
http://download.journals.elsevierhealth.com/pdfs/journals/1083-8791/PIIS1083879107001437.pdf

Chronic graft-versus host disease (GVHD) in children with mucopolysaccharidoses (MPS) two years after unrelated donor umbilical cord blood transplantation (UCBT). Szabolcs, P., Martin, R. L., Parikh, S., Prasad, V. K., Vinesett, R., Escolar, M. L., et al. (2007). Biol Blood Marrow Transplant, 13(2), 67-67.

Neurophysiologic assessment of mucopolysaccharidosis III. Husain, A. M., Escolar, M. L., & Kurtzberg, J. (2006). Clin Neurophysiol, 117(9), 2059-2063.
http://www.clinph-journal.com/article/S1388-2457(06)00260-4/abstract

Gross motor abilities in children with Hurler syndrome. Dusing, S. C., Thorpe, D., Rosenberg, A., Mercer, V., & Escolar, M. L. (2006). Dev Med Child Neurol, 48(11), 927-930.
http://onlinelibrary.wiley.com/store/10.1111/j.1469-8749.2006.02027a.x/asset/j.1469-8749.2006.02027a.x.pdf?v=1&t=hfopogno&s=0161f7669954bf5e0eed7e02b56bf971ce5b4db9

Neonatal umbilical cord blood transplantation favorably alters the neurodevelopmental outcome and survival of babies with infantile Krabbe disease. Wood, S. J., Allison, J., Escolar, M. L., Provenzale, J. M., & Kurtzberg, J. (2006). Biol Blood Marrow Transplant, 12(2), 153-154.
http://www.clinph-journal.com/article/S1388-2457(06)00260-4/abstract

Neurodevelopmental outcome of children with infantile Krabbe disease treated with umbilical cord blood transplantation: 10 years of follow-up.
Escolar, M. L., Yelin, K., & Poe, M. D. (2006). Lysosomal Storage Diseases, 6(3), 71-79.
http://connection.ebscohost.com/c/articles/24208288/neurodevelopmental-outcomes-children-infantile-krabbe-disease-treated-umbilical-cord-blood-transplantation-10-years-follow-up

Neurobehavioral outcome in children with MPS III (Sanfilippo Syndrome) after unrelated donor umbilical cord blood transplantation (UCBT). Escolar, Lakshminayanaran, Szabolcs, Poe, Prasad, Parikh, et al. (2006). Biol Blood Marrow Transplant, 12(2), 125-125.

Gross and fine motor skills of children with Hurler syndrome (MPS-IH) post umbilical cord blood transplantation: a case series report. Dusing, S. C., Rosenberg, A., Hiemenz, J. R., Piner, S., & Escolar, M. (2005). Pediatr Phys Ther, 17(4), 264-267.
http://journals.lww.com/pedpt/Fulltext/2005/01740/Gross_and_Fine_Motor_Skills_of_Children_with.6.aspx

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. Escolar, M. L., Poe, M. D., Provenzale, J. M., Richards, K. C., Allison, J., Wood, S., et al. (2005). Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med, 352(20), 2069-2081.
http://www.nejm.org/doi/pdf/10.1056/NEJMoa042604

Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience. McGraw, P., Liang, L., Escolar, M., Mukundan, S., Kurtzberg, J., & Provenzale, J. M. (2005). Radiology, 236(1), 221-230.
http://radiology.rsna.org/content/236/1/221.long

Treatment of pediatric patients with Sanfilippo syndrome (MPS IIIA and IIIB) with unrelated umbilical cord blood transplantation. Kurtzberg, J., Szabolcs, P., Wood, S., Ciocci, G., Driscoll, T., Prasad, V., et al. (2005). Biol Blood Marrow Transplant, 11(2), 83-84.

Quantitative analysis of diffusion tensor imaging data in serial assessment of Krabbe disease. Provenzale, J. M., Escolar, M., & Kurtzberg, J. (2005). Ann N Y Acad Sci, 1064, 220-229.
http://onlinelibrary.wiley.com/doi/10.1196/annals.1340.040/abstract

Transplant outcomes and stabilization of neurological function in young children with MPS III (Sanfilippo syndrome) after unrelated donor umbilical cord blood transplantation. Lakshminarayanan, S., Szabolcs, P., Prasad, V. K., Parikh, S., Allison, June, W., Susan J. , Escolar, M., et al. (2005).
Paper presented at the ASH Annual Meeting Abstracts 2005

Purine nucleoside phosphorylase deficiency (PNP-def) presenting with lymphopenia and developmental delay: successful correction with umbilical cord blood transplantation. Myers, L. A., Hershfield, M. S., Neale, W. T., Escolar, M., Kurtzberg, J. (2004). J Pediatr, 145(5), 710-712.
(link to http://www.jpeds.com/article/S0022-3476(04)00641-9/abstract)

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. Staba, S. L., Escolar, M. L., Poe, M., Kim, Y., Martin, P. L., Szabolcs, P., et al. (2004). N Engl J Med, 350(19), 1960-1969.
http://www.nejm.org/doi/pdf/10.1056/NEJMoa032613

When Hearing Loss Occurs with Multiple Disabilities. Roush, J. P. D., Holcomb, M. B. A., Roush, P. M. A., & Escolar, M. L. M. D. (2004). Seminars in Hearing (04), 333-345.
https://www.thieme-connect.com/ejournals/abstract/10.1055/s-2004-836135

Preliminary results of unrelated umbilical cord blood transplantation for Sanfilippo syndrome (MPS III). Staba, S. L., Escolar, M. L., Provenzale, J. M., Wood, S., Allison, J., Cash, J., et al. (2003). Blood, 102(11), 481A-481A.

Unrelated donor umbilical cord blood transplantation for X-linked adrenoleukodystrophy. Kurtzberg, J., Szabolcs, P., Martin, P. L., Driscoll, T., Kelly, T., Ciocci, G., et al. (2003). Blood, 102(11), 479B-479B.

Unrelated umbilical cord blood transplantation for hurler syndrome. Staba, Escolar, Kim, Martin, Szabolcs, Mustafa, et al. (2003). Biol Blood Marrow Transplant, 9(2), 63-63.

Purine nucleoside phosphorylase deficiency presenting with lymphopenia and hypogammaglobulinemia. Myers, L. A., Neale, W. T., Hershfield, M. S., & Escolar, M. L. (2001). J Allergy Clin Immun, 107(2), S204-S204.

 
Please contact the NDRD team for reprints of any of these publications.