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Items: 1 to 50 of 177

1.

Cellular Therapy in Rare Childhood Neurologic Disease: Lessons, Outcomes, and Access.

Orchard PJ.

J Child Neurol. 2018 Sep 11:883073818797875. doi: 10.1177/0883073818797875. [Epub ahead of print] No abstract available.

PMID:
30203711
2.

Genome-wide association study of response to methotrexate in early rheumatoid arthritis patients.

Taylor JC, Bongartz T, Massey J, Mifsud B, Spiliopoulou A, Scott IC, Wang J, Morgan M, Plant D, Colombo M, Orchard P, Twigg S, McInnes IB, Porter D, Freeston JE, Nam JL, Cordell HJ, Isaacs JD, Strathdee JL, Arnett D, de Hair MJH, Tak PP, Aslibekyan S, van Vollenhoven RF, Padyukov L, Bridges SL, Pitzalis C, Cope AP, Verstappen SMM, Emery P, Barnes MR, Agakov F, McKeigue P, Mushiroda T, Kubo M, Weinshilboum R, Barton A, Morgan AW, Barrett JH; MATURA; and PAMERA; Consortia.

Pharmacogenomics J. 2018 Jul;18(4):528-538. doi: 10.1038/s41397-018-0025-5. Epub 2018 May 25. Review.

PMID:
29795407
3.

CNS Langerhans cell histiocytosis: Common hematopoietic origin for LCH-associated neurodegeneration and mass lesions.

McClain KL, Picarsic J, Chakraborty R, Zinn D, Lin H, Abhyankar H, Scull B, Shih A, Lim KPH, Eckstein O, Lubega J, Peters TL, Olea W, Burke T, Ahmed N, Hicks MJ, Tran B, Jones J, Dauser R, Jeng M, Baiocchi R, Schiff D, Goldman S, Heym KM, Wilson H, Carcamo B, Kumar A, Rodriguez-Galindo C, Whipple NS, Campbell P, Murdoch G, Kofler J, Heales S, Malone M, Woltjer R, Quinn JF, Orchard P, Kruer MC, Jaffe R, Manz MG, Lira SA, Parsons DW, Merad M, Man TK, Allen CE.

Cancer. 2018 Jun 15;124(12):2607-2620. doi: 10.1002/cncr.31348. Epub 2018 Apr 6.

PMID:
29624648
4.

Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy.

Pierpont EI, McCoy E, King KE, Ziegler RS, Shanley R, Nascene D, Raymond GV, Phelan R, Lund TC, Orchard PJ, Miller WP.

Ann Clin Transl Neurol. 2018 Jan 22;5(3):252-261. doi: 10.1002/acn3.526. eCollection 2018 Mar.

5.

Publisher Correction: Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1.

Makino E, Klodnitsky H, Leonard J, Lillie J, Lund TC, Marshall J, Nietupski J, Orchard PJ, Miller WP, Phaneuf C, Tietz D, Varban ML, Donovan M, Belenki A.

Sci Rep. 2018 Mar 19;8(1):4994. doi: 10.1038/s41598-018-23332-3.

6.

Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.

Eisengart JB, Rudser KD, Xue Y, Orchard P, Miller W, Lund T, Van der Ploeg A, Mercer J, Jones S, Mengel KE, Gökce S, Guffon N, Giugliani R, de Souza CFM, Shapiro EG, Whitley CB.

Genet Med. 2018 Mar 8. doi: 10.1038/gim.2018.29. [Epub ahead of print]

7.

Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1.

Makino E, Klodnitsky H, Leonard J, Lillie J, Lund TC, Marshall J, Nietupski J, Orchard PJ, Miller WP, Phaneuf C, Tietz D, Varban ML, Donovan M, Belenki A.

Sci Rep. 2018 Feb 27;8(1):3681. doi: 10.1038/s41598-018-22078-2. Erratum in: Sci Rep. 2018 Mar 19;8(1):4994.

8.

Metabolic Syndrome and Cardiovascular Risk Factors after Hematopoietic Cell Transplantation in Severe Mucopolysaccharidosis Type I (Hurler Syndrome).

Braunlin E, Steinberger J, DeFor T, Orchard P, Kelly AS.

Biol Blood Marrow Transplant. 2018 Jun;24(6):1289-1293. doi: 10.1016/j.bbmt.2018.01.028. Epub 2018 Feb 1.

PMID:
29409846
9.

Successful hematopoietic cell transplantation following cardiac transplantation in two pediatric patients.

Phelan R, Smith AR, Orchard PJ, Ameduri RK.

Pediatr Transplant. 2018 Mar;22(2). doi: 10.1111/petr.13103. Epub 2018 Jan 19.

PMID:
29352516
10.

Treatment of brain disease in the mucopolysaccharidoses.

Scarpa M, Orchard PJ, Schulz A, Dickson PI, Haskins ME, Escolar ML, Giugliani R.

Mol Genet Metab. 2017 Dec;122S:25-34. doi: 10.1016/j.ymgme.2017.10.007. Epub 2017 Oct 16. Review.

11.

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.

N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.

12.

Quality of life among boys with adrenoleukodystrophy following hematopoietic stem cell transplant.

Beckmann NB, Miller WP, Dietrich MS, Orchard PJ.

Child Neuropsychol. 2018 Oct;24(7):986-998. doi: 10.1080/09297049.2017.1380176. Epub 2017 Sep 21.

PMID:
28934891
13.

Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis.

Hinderer C, Katz N, Louboutin JP, Bell P, Tolar J, Orchard PJ, Lund TC, Nayal M, Weng L, Mesaros C, de Souza CFM, Dalla Corte A, Giugliani R, Wilson JM.

Hum Mol Genet. 2017 Oct 1;26(19):3837-3849. doi: 10.1093/hmg/ddx277.

PMID:
28934395
14.

Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients.

Ivaturi V, Dvorak CC, Chan D, Liu T, Cowan MJ, Wahlstrom J, Stricherz M, Jennissen C, Orchard PJ, Tolar J, Pai SY, Huang L, Aweeka F, Long-Boyle J.

Biol Blood Marrow Transplant. 2017 Oct;23(10):1701-1713. doi: 10.1016/j.bbmt.2017.06.021. Epub 2017 Jul 3.

15.

Diagnosis and Management of Osteopetrosis: Consensus Guidelines From the Osteopetrosis Working Group.

Wu CC, Econs MJ, DiMeglio LA, Insogna KL, Levine MA, Orchard PJ, Miller WP, Petryk A, Rush ET, Shoback DM, Ward LM, Polgreen LE.

J Clin Endocrinol Metab. 2017 Sep 1;102(9):3111-3123. doi: 10.1210/jc.2017-01127. Review.

PMID:
28655174
16.

Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: a retrospective analysis.

Eapen M, Wang T, Veys PA, Boelens JJ, St Martin A, Spellman S, Bonfim CS, Brady C, Cant AJ, Dalle JH, Davies SM, Freeman J, Hsu KC, Fleischhauer K, Kenzey C, Kurtzberg J, Michel G, Orchard PJ, Paviglianiti A, Rocha V, Veneris MR, Volt F, Wynn R, Lee SJ, Horowitz MM, Gluckman E, Ruggeri A.

Lancet Haematol. 2017 Jul;4(7):e325-e333. doi: 10.1016/S2352-3026(17)30104-7. Epub 2017 Jun 13.

17.

The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison.

Wyffels ML, Orchard PJ, Shanley RM, Miller WP, Van Heest AE.

J Hand Surg Am. 2017 Jul;42(7):573.e1-573.e8. doi: 10.1016/j.jhsa.2017.03.036. Epub 2017 May 4.

PMID:
28479223
18.

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.

Belur LR, Temme A, Podetz-Pedersen KM, Riedl M, Vulchanova L, Robinson N, Hanson LR, Kozarsky KF, Orchard PJ, Frey WH 2nd, Low WC, McIvor RS.

Hum Gene Ther. 2017 Jul;28(7):576-587. doi: 10.1089/hum.2017.187. Epub 2017 Apr 20.

19.

Neurocognitive Trajectory of Boys Who Received a Hematopoietic Stem Cell Transplant at an Early Stage of Childhood Cerebral Adrenoleukodystrophy.

Pierpont EI, Eisengart JB, Shanley R, Nascene D, Raymond GV, Shapiro EG, Ziegler RS, Orchard PJ, Miller WP.

JAMA Neurol. 2017 Jun 1;74(6):710-717. doi: 10.1001/jamaneurol.2017.0013.

20.

Design and Validation of a Three-Dimensional Printed Flexible Canine Otoscopy Teaching Model.

Nibblett BMD, Pereira MM, Sithole F, Orchard PAD, Bauman EB.

Simul Healthc. 2017 Apr;12(2):91-95. doi: 10.1097/SIH.0000000000000227.

21.

GeneImp: Fast Imputation to Large Reference Panels Using Genotype Likelihoods from Ultralow Coverage Sequencing.

Spiliopoulou A, Colombo M, Orchard P, Agakov F, McKeigue P.

Genetics. 2017 May;206(1):91-104. doi: 10.1534/genetics.117.200063. Epub 2017 Mar 27.

22.

Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome.

Lum SH, Miller WP, Jones S, Poulton K, Ogden W, Lee H, Logan A, Bonney D, Lund TC, Orchard PJ, Wynn RF.

Bone Marrow Transplant. 2017 Jun;52(6):846-853. doi: 10.1038/bmt.2017.5. Epub 2017 Feb 20.

PMID:
28218755
23.

Genetic regulatory signatures underlying islet gene expression and type 2 diabetes.

Varshney A, Scott LJ, Welch RP, Erdos MR, Chines PS, Narisu N, Albanus RD, Orchard P, Wolford BN, Kursawe R, Vadlamudi S, Cannon ME, Didion JP, Hensley J, Kirilusha A; NISC Comparative Sequencing Program, Bonnycastle LL, Taylor DL, Watanabe R, Mohlke KL, Boehnke M, Collins FS, Parker SC, Stitzel ML.

Proc Natl Acad Sci U S A. 2017 Feb 28;114(9):2301-2306. doi: 10.1073/pnas.1621192114. Epub 2017 Feb 13.

24.

Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature.

Mallhi K, Orchard PJ, Miller WP, Cao Q, Tolar J, Lund TC.

Bone Marrow Transplant. 2017 May;52(5):726-732. doi: 10.1038/bmt.2016.356. Epub 2017 Jan 16.

25.

Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience.

Rodgers NJ, Kaizer AM, Miller WP, Rudser KD, Orchard PJ, Braunlin EA.

J Inherit Metab Dis. 2017 Mar;40(2):271-280. doi: 10.1007/s10545-016-0006-2. Epub 2017 Jan 4.

PMID:
28054207
26.

Allele-Level HLA Matching Impacts Key Outcomes Following Umbilical Cord Blood Transplantation for Inherited Metabolic Disorders.

Mallhi KK, Smith AR, DeFor TE, Lund TC, Orchard PJ, Miller WP.

Biol Blood Marrow Transplant. 2017 Jan;23(1):119-125. doi: 10.1016/j.bbmt.2016.10.019. Epub 2016 Oct 29.

27.

Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H.

Raymond GV, Pasquali M, Polgreen LE, Dickson PI, Miller WP, Orchard PJ, Lund TC.

Sci Rep. 2016 Dec 2;6:38305. doi: 10.1038/srep38305.

28.

Haploidentical transplantation with post-transplant cyclophosphamide following reduced-intensity conditioning for osteopetrosis: outcomes in three children.

Bahr TL, Lund T, Sando NM, Orchard PJ, Miller WP.

Bone Marrow Transplant. 2016 Nov;51(11):1546-1548. doi: 10.1038/bmt.2016.180. Epub 2016 Jul 4. No abstract available.

PMID:
27376448
29.

Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues.

Hendriksz CJ, Berger KI, Lampe C, Kircher SG, Orchard PJ, Southall R, Long S, Sande S, Gold JI.

Orphanet J Rare Dis. 2016 Aug 26;11(1):119. doi: 10.1186/s13023-016-0503-2. Review.

30.

Glycosylation of plasma IgG in colorectal cancer prognosis.

Theodoratou E, Thaçi K, Agakov F, Timofeeva MN, Štambuk J, Pučić-Baković M, Vučković F, Orchard P, Agakova A, Din FV, Brown E, Rudd PM, Farrington SM, Dunlop MG, Campbell H, Lauc G.

Sci Rep. 2016 Jun 15;6:28098. doi: 10.1038/srep28098.

31.

Unique medical issues in adult patients with mucopolysaccharidoses.

Mitchell J, Berger KI, Borgo A, Braunlin EA, Burton BK, Ghotme KA, Kircher SG, Molter D, Orchard PJ, Palmer J, Pastores GM, Rapoport DM, Wang RY, White K.

Eur J Intern Med. 2016 Oct;34:2-10. doi: 10.1016/j.ejim.2016.05.017. Epub 2016 Jun 11. Review.

32.

Rapid Induction of Cerebral Organoids From Human Induced Pluripotent Stem Cells Using a Chemically Defined Hydrogel and Defined Cell Culture Medium.

Lindborg BA, Brekke JH, Vegoe AL, Ulrich CB, Haider KT, Subramaniam S, Venhuizen SL, Eide CR, Orchard PJ, Chen W, Wang Q, Pelaez F, Scott CM, Kokkoli E, Keirstead SA, Dutton JR, Tolar J, O'Brien TD.

Stem Cells Transl Med. 2016 Jul;5(7):970-9. doi: 10.5966/sctm.2015-0305. Epub 2016 May 13.

33.

Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy.

Thibert KA, Raymond GV, Tolar J, Miller WP, Orchard PJ, Lund TC.

Sci Rep. 2016 Apr 15;6:24579. doi: 10.1038/srep24579.

34.

Progression of Hip Dysplasia in Mucopolysaccharidosis Type I Hurler After Successful Hematopoietic Stem Cell Transplantation.

Langereis EJ, den Os MM, Breen C, Jones SA, Knaven OC, Mercer J, Miller WP, Kelly PM, Kennedy J, Ketterl TG, O'Meara A, Orchard PJ, Lund TC, van Rijn RR, Sakkers RJ, White KK, Wijburg FA.

J Bone Joint Surg Am. 2016 Mar 2;98(5):386-95. doi: 10.2106/JBJS.O.00601.

PMID:
26935461
35.

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres.

Ghosh A, Miller W, Orchard PJ, Jones SA, Mercer J, Church HJ, Tylee K, Lund T, Bigger BW, Tolar J, Wynn RF.

Mol Genet Metab. 2016 Mar;117(3):373-7. doi: 10.1016/j.ymgme.2016.01.011. Epub 2016 Jan 27.

36.

Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.

Kunin-Batson AS, Shapiro EG, Rudser KD, Lavery CA, Bjoraker KJ, Jones SA, Wynn RF, Vellodi A, Tolar J, Orchard PJ, Wraith JE.

JIMD Rep. 2016;29:95-102. Epub 2016 Jan 30.

37.

Anatomy of the bacitracin resistance network in Bacillus subtilis.

Radeck J, Gebhard S, Orchard PS, Kirchner M, Bauer S, Mascher T, Fritz G.

Mol Microbiol. 2016 May;100(4):607-20. doi: 10.1111/mmi.13336. Epub 2016 Mar 10.

38.

Mechanisms of Antioxidant Induction with High-Dose N-Acetylcysteine in Childhood Cerebral Adrenoleukodystrophy.

Kartha RV, Zhou J, Basso L, Schröder H, Orchard PJ, Cloyd J.

CNS Drugs. 2015 Dec;29(12):1041-7. doi: 10.1007/s40263-015-0300-9.

PMID:
26670322
39.

How Effective Is Initial Military-Specific Training in the Development of Physical Performance of Soldiers?

Groeller H, Burley S, Orchard P, Sampson JA, Billing DC, Linnane D.

J Strength Cond Res. 2015 Nov;29 Suppl 11:S158-62. doi: 10.1519/JSC.0000000000001066.

PMID:
26506181
40.

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.

AJNR Am J Neuroradiol. 2016 Feb;37(2):367-72. doi: 10.3174/ajnr.A4500. Epub 2015 Oct 1.

41.

Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation.

Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Savani BN, Veys PA, Bredeson CN, Giralt SA, LeMaistre CF.

Biol Blood Marrow Transplant. 2015 Nov;21(11):1863-1869. doi: 10.1016/j.bbmt.2015.07.032. Epub 2015 Aug 7.

42.

Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.

Boucher AA, Miller W, Shanley R, Ziegler R, Lund T, Raymond G, Orchard PJ.

Orphanet J Rare Dis. 2015 Aug 7;10:94. doi: 10.1186/s13023-015-0313-y.

43.

Second allogeneic hematopoietic cell transplantation for graft failure: poor outcomes for neutropenic graft failure.

Lund TC, Liegel J, Bejanyan N, Orchard PJ, Cao Q, Tolar J, Brunstein C, Wagner JE, Verneris MR, Weisdorf D.

Am J Hematol. 2015 Oct;90(10):892-6. doi: 10.1002/ajh.24111.

44.

Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.

Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB.

Mol Genet Metab. 2015 Sep-Oct;116(1-2):61-8. doi: 10.1016/j.ymgme.2015.06.002. Epub 2015 Jun 17.

45.

Hematopoietic stem cell transplantation for infantile osteopetrosis.

Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, Al-Seraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Perez MA, Veys PA, Eapen M.

Blood. 2015 Jul 9;126(2):270-6. doi: 10.1182/blood-2015-01-625541. Epub 2015 May 26.

46.

Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning.

Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M.

Br J Haematol. 2015 Jun;169(5):711-8. doi: 10.1111/bjh.13347. Epub 2015 Mar 27.

47.

Long-Term Results of Carpal Tunnel and Trigger Finger Releases in a Patient with Hurler Syndrome.

Wyffels M, Orchard PJ, Van Heest AE.

JBJS Case Connect. 2015 Mar 25;5(1):e27. doi: 10.2106/JBJS.CC.N.00132.

PMID:
29252605
48.

Minimally invasive component separation techniques in complex ventral abdominal hernia repair: a systematic review of the literature.

Feretis M, Orchard P.

Surg Laparosc Endosc Percutan Tech. 2015 Apr;25(2):100-5. doi: 10.1097/SLE.0000000000000114. Review.

PMID:
25793353
49.

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.

Aldenhoven M, Wynn RF, Orchard PJ, O'Meara A, Veys P, Fischer A, Valayannopoulos V, Neven B, Rovelli A, Prasad VK, Tolar J, Allewelt H, Jones SA, Parini R, Renard M, Bordon V, Wulffraat NM, de Koning TJ, Shapiro EG, Kurtzberg J, Boelens JJ.

Blood. 2015 Mar 26;125(13):2164-72. doi: 10.1182/blood-2014-11-608075. Epub 2015 Jan 26.

50.

Long-Term Results of Carpal Tunnel and Trigger Finger Releases in a Patient with Hurler Syndrome: A Case Report.

Wyffels M, Orchard PJ, Van Heest AE.

JBJS Case Connect. 2015 Jan-Mar;5(1):e27. doi: 10.2106/JBJS.CC.N.00132.

PMID:
29252733

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