Works matching DE "MUCOPOLYSACCHARIDOSIS treatment"

Results: 71

Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005.

Published in:

Bone Marrow Transplantation, 2009, v. 43, n. 5, p. 375, doi. 10.1038/bmt.2008.328

By:

Sauer, M.;
Meissner, B.;
Fuchs, D.;
Gruhn, B.;
Kabisch, H.;
Erttmann, R.;
Suttorp, M.;
Beilken, A.;
Luecke, T.;
Welte, K.;
Grigull, L.;
Sykora, K. W.

Publication type:

Article

Mucopolysaccharidosis VI: the Italian experience.

Published in:

2009

By:

Scarpa, Maurizio;
Barone, Rita;
Fiumara, Agata;
Astarita, Luca;
Parenti, Giancarlo;
Rampazzo, Angelica;
Sala, Stefania;
Sorge, Giovanni;
Parini, Rossella

Publication type:

journal article

A new case of mucopolysaccharidosis VII presenting as non immune hydrops fetalis.

Published in:

European Journal of Pediatrics, 2003, v. 162, n. 7/8, p. 520, doi. 10.1007/s00431-003-1198-7

By:

Walter-Nicolet, Elisabeth;
Rakza, Thameur;
Storme, Laurent;
Vaillant, Catherine;
Magnenant, Eric;
Cremer, Robin;
Thumerelle, Caroline;
Dobbelaere, Dries

Publication type:

Article

Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments.

Published in:

Drugs, 2019, v. 79, n. 10, p. 1103, doi. 10.1007/s40265-019-01147-4

By:

Sawamoto, Kazuki;
Stapleton, Molly;
Alméciga-Díaz, Carlos J.;
Espejo-Mojica, Angela J.;
Losada, Juan Camilo;
Suarez, Diego A.;
Tomatsu, Shunji

Publication type:

Article

Update on mucopolysaccharidosis type II.

Published in:

Acta Paediatrica, 2007, p. 55, doi. 10.1111/j.1651-2227.2007.00210.x

By:

Beck, Michael;
Wraith, Ed

Publication type:

Article

Miscellaneous.

Published in:: Current Medical Literature: Growth, Growth Hormone, & Metabolism, 2010, v. 3, n. 1, p. 19
Publication type:: Article

Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure.

Published in:

2019

By:

Kuiper, Gé-Ann;
Langereis, Eveline J.;
Breyer, Sandra;
Carbone, Marco;
Castelein, René M.;
Eastwood, Deborah M.;
Garin, Christophe;
Guffon, Nathalie;
van Hasselt, Peter M.;
Hensman, Pauline;
Jones, Simon A.;
Kenis, Vladimir;
Kruyt, Moyo;
van der Lee, Johanna H.;
Mackenzie, William G.;
Orchard, Paul J.;
Oxborrow, Neil;
Parini, Rossella;
Robinson, Amy;
Schubert Hjalmarsson, Elke

Publication type:

journal article

Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA.

Published in:

2018

By:

Lin, Hsiang-Yu;
Chen, Ming-Ren;
Lin, Shan-Miao;
Hung, Chung-Lieh;
Niu, Dau-Ming;
Chuang, Chih-Kuang;
Lin, Shuan-Pei

Publication type:

journal article

Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI.

Published in:

PLoS ONE, 2011, v. 6, n. 8, p. 1, doi. 10.1371/journal.pone.0022447

By:

Eliyahu, Efrat;
Wolfson, Theodore;
Ge, Yi;
Jepsen, Karl J.;
Schuchman, Edward H.;
Simonaro, Calogera M.

Publication type:

Article

Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice.

Published in:

PLoS ONE, 2010, v. 5, n. 8, p. 1, doi. 10.1371/journal.pone.0012194

By:

Dvorak-Ewell, Melita;
Wendt, Dan;
Hague, Chuck;
Christianson, Terri;
Koppaka, Vish;
Crippen, Danielle;
Kakkis, Emil;
Vellard, Michel

Publication type:

Article

Carpal tunnel syndrome in mucopolysaccharidosis: the value of rare disease registries.

Published in:

2017

By:

White, Klane K

Publication type:

journal article

Drug Repurposing Patent Applications April-June 2015.

Published in:

Assay & Drug Development Technologies, 2015, v. 13, n. 10, p. 654, doi. 10.1089/adt.2015.29030.pq2

By:

Mucke, Hermann A.M.

Publication type:

Article

Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome.

Published in:

Journal of Patient-Reported Outcomes, 2022, v. 6, n. 1, p. 1, doi. 10.1186/s41687-022-00447-w

By:

Porter, Katherine Ackerman;
O'Neill, Cara;
Drake, Elise;
Andrews, Sara M.;
Delaney, Kathleen;
Parker, Samantha;
Escolar, Maria L.;
Montgomery, Stacey;
Moon, William;
Worrall, Carolyn;
Peay, Holly L.

Publication type:

Article

Regression Trends & Treatment Effectiveness to Improve Quality of Life for a Pre-Adolescent Girl with MPS IIIA.

Published in:

Journal of Developmental & Physical Disabilities, 2018, v. 30, n. 4, p. 545, doi. 10.1007/s10882-018-9601-5

By:

Schreck, Kimberly A.;
Helsel, Cortney;
Paxon, Alisha;
Weston, Kasey;
Daniels, Mia

Publication type:

Article

Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation.

Published in:

Bone Marrow Transplantation, 1998, v. 21, n. 6, p. 629, doi. 10.1038/sj.bmt.1701141

By:

Yamada, Y;
Kato, K;
Sukegawa, K;
Tomatsu, S;
Fukuda, S;
Emura, S;
Kojima, S;
Matsuyama, T;
Sly, W S;
Kondo, N;
Orii, T

Publication type:

Article

Treatment of MPS I mice with microencapsulated cells overexpressing IDUA: effect of the prednisolone administration.

Published in:

Journal of Microencapsulation, 2013, v. 30, n. 4, p. 383, doi. 10.3109/02652048.2012.746745

By:

Lagranha, Valeska Lizzi;
de Carvalho, Talita Giacomet;
Giugliani, Roberto;
Matte, Ursula

Publication type:

Article

Sanfilippo syndrome: consensus guidelines for clinical care.

Published in:

2022

By:

Muschol, Nicole;
Giugliani, Roberto;
Jones, Simon A.;
Muenzer, Joseph;
Smith, Nicholas J. C.;
Whitley, Chester B.;
Donnell, Megan;
Drake, Elise;
Elvidge, Kristina;
Melton, Lisa;
O'Neill, Cara;
MPS III Guideline Development Group

Publication type:

journal article

Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.

Published in:

2018

By:

Holley, Rebecca J.;
Ellison, Stuart M.;
Fil, Daniel;
O'Leary, Claire;
McDermott, John;
Senthivel, Nishanthi;
Langford-Smith, Alexander W. W.;
Wilkinson, Fiona L.;
D'Souza, Zelpha;
Parker, Helen;
Liao, Aiyin;
Rowlston, Samuel;
Gleitz, Hélène F. E.;
Kan, Shih-Hsin;
Dickson, Patricia I.;
Bigger, Brian W.

Publication type:

journal article

Sanfilippo syndrome: Overall review.

Published in:

Pediatrics International, 2015, v. 57, n. 3, p. 331, doi. 10.1111/ped.12636

By:

Andrade, Fernando;
Aldámiz‐Echevarría, Luis;
Llarena, Marta;
Couce, María Luz

Publication type:

Article

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.

Published in:

2011

By:

de Ru, Minke H;
Boelens, Jaap J;
Das, Anibh M;
Jones, Simon A;
van der Lee, Johanna H;
Mahlaoui, Nizar;
Mengel, Eugen;
Offringa, Martin;
O'Meara, Anne;
Parini, Rossella;
Rovelli, Attilio;
Sykora, Karl-Walter;
Valayannopoulos, Vassili;
Vellodi, Ashok;
Wynn, Robert F;
Wijburg, Frits A

Publication type:

journal article

Does enzyme replacement therapy influence the ocular changes in type VI mucopolysaccharidosis?

Published in:

Graefe's Archive of Clinical & Experimental Ophthalmology, 2009, v. 247, n. 7, p. 975, doi. 10.1007/s00417-008-1030-1

By:

Susanne Pitz;
Olufunmilola Ogun;
Laila Arash;
Elke Miebach;
Michael Beck

Publication type:

Article

Ocular manifestations and management recommendations of lysosomal storage disorders I: mucopolysaccharidoses.

Published in:

Clinical Ophthalmology, 2015, v. 9, p. 1633, doi. 10.2147/OPTH.S78368

By:

Fenzl, Carlton R.;
Kyla Teramoto;
Moshirfar, Majid

Publication type:

Article

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

Published in:

Molecular Therapy, 2015, v. 23, n. 8, p. 1298, doi. 10.1038/mt.2015.99

By:

Hinderer, Christian;
Bell, Peter;
Louboutin, Jean-Pierre;
Zhu, Yanqing;
Yu, Hongwei;
Lin, Gloria;
Choa, Ruth;
Gurda, Brittney L;
Bagel, Jessica;
O'Donnell, Patricia;
Sikora, Tracey;
Ruane, Therese;
Wang, Ping;
Tarantal, Alice F;
Casal, Margret L;
Haskins, Mark E;
Wilson, James M

Publication type:

Article

Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome.

Published in:

Molecular Therapy, 2010, v. 18, n. 6, p. 1094, doi. 10.1038/mt.2010.32

By:

Tomatsu, Shunji;
Montaño, Adriana M.;
Dung, Vu Chi;
Ohashi, Amiko;
Oikawa, Hirotaka;
Oguma, Toshihiro;
Orii, Tadao;
Barrera, Luis;
Sly, William S.

Publication type:

Article

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-L-iduronidase Fusion Gene Product.

Published in:

Molecular Therapy, 2008, v. 16, n. 8, p. 1459, doi. 10.1038/mt.2008.119

By:

Osborn, Mark J.;
McElmurry, Ron T.;
Peacock, Brandon;
Tolar, Jakub;
Blazar, Bruce R.

Publication type:

Article

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.

Published in:

Current Medical Research & Opinion, 2009, v. 25, n. 5, p. 1285, doi. 10.1185/03007990902892633

By:

M. Schlander;
M. Beck

Publication type:

Article

Laronidase.

Published in:: BioDrugs, 2002, v. 16, n. 4, p. 316, doi. 10.2165/00063030-200216040-00009
Publication type:: Article

Anaesthetic implications of the changing management of patients with mucopolysaccharidosis.

Published in:

2016

By:

Hack, H. A.;
Walker, R. W. M.;
Gardiner, P.;
Walker, Rwm

Publication type:

journal article

Anesthetic care and perioperative complications of children with Morquio syndrome.

Published in:

Pediatric Anesthesia, 2012, v. 22, n. 9, p. 901, doi. 10.1111/j.1460-9592.2012.03904.x

By:

Theroux, Mary C.;
Nerker, Tanvi;
Ditro, Colleen;
Mackenzie, William G.

Publication type:

Article

Hurler Syndrome: A report of three cases.

Published in:

International Archives of Integrated Medicine, 2015, v. 2, n. 6, p. 210

By:

Makhela J.;
Kaushal K.;
Choraria S.;
Ranka M.;
Patel S. B.

Publication type:

Article

Abstract- Poster.

Published in:: 2022
Publication type:: Abstract

Sanfilippo syndrome.

Published in:

Learning Disability Practice, 2019, v. 22, n. 4, p. 17, doi. 10.7748/ldp.22.4.17.s15

By:

Hall, Sarah;
Henderson-Laidlaw, Mhari

Publication type:

Article

Treatment for Rare Enzyme Disorder.

Published in:

JAMA: Journal of the American Medical Association, 2018, v. 319, n. 1, p. 14, doi. 10.1001/jama.2017.20058

By:

Voelker, Rebecca

Publication type:

Article

NDST1 Preferred Promoter Confirmation and Identification of Corresponding Transcriptional Inhibitors as Substrate Reduction Agents for Multiple Mucopolysaccharidosis Disorders.

Published in:

PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162145

By:

Tkachyova, Ilona;
Fan, Xiaolian;
LamHonWah, Anne-Marie;
Fedyshyn, Bohdana;
Tein, Ingrid;
Mahuran, Don J.;
Schulze, Andreas

Publication type:

Article

Heart and Cardiovascular Involvement in Patients with Mucopolysaccharidosis Type IVA (Morquio-A Syndrome).

Published in:

PLoS ONE, 2016, v. 11, n. 9, p. 1, doi. 10.1371/journal.pone.0162612

By:

Kampmann, Christoph;
Abu-Tair, Tariq;
Gökce, Seyfullah;
Lampe, Christina;
Reinke, Jörg;
Mengel, Eugen;
Hennermann, Julia B.;
Wiethoff, Christiane M.

Publication type:

Article

Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs.

Published in:

PLoS ONE, 2016, v. 11, n. 4, p. 1, doi. 10.1371/journal.pone.0153136

By:

Simonaro, Calogera M.;
Tomatsu, Shunji;
Sikora, Tracy;
Kubaski, Francyne;
Frohbergh, Michael;
Guevara, Johana M.;
Wang, Raymond Y.;
Vera, Moin;
Kang, Jennifer L.;
Smith, Lachlan J.;
Schuchman, Edward H.;
Haskins, Mark E.

Publication type:

Article

Elosulfase alfa for the treatment of mucopolysacchoridosis IVA.

Published in:

Expert Review of Endocrinology & Metabolism, 2015, v. 10, n. 6, p. 569, doi. 10.1586/17446651.2015.1091723

By:

Tanpaiboon, Pranoot

Publication type:

Article

Mucopolysaccharidosis IIIB and mild skeletal anomalies: coexistence of <italic>NAGLU</italic> and <italic>CYP26B1</italic> missense variations in the same patient in a Chinese family.

Published in:

BMC Medical Genetics, 2018, v. 19, n. 1, p. 1, doi. 10.1186/s12881-018-0562-4

By:

Li, Jinliang;
Xie, Han;
Jiang, Yuwu

Publication type:

Article

Mucopolysaccharidosis: A broad review.

Published in:

2022

By:

Nagpal, Ritu;
Goyal, Ram;
Priyadarshini, K;
Kashyap, Seema;
Sharma, Mohita;
Sinha, Rajesh;
Sharma, Namrata;
Goyal, Ram Bharos

Publication type:

journal article

Combined spinal arthrodesis with instrumentation for the management of progressive thoracolumbar kyphosis in children with mucopolysaccharidosis.

Published in:

European Spine Journal, 2014, v. 23, n. 12, p. 2751, doi. 10.1007/s00586-014-3186-1

By:

Garrido, Enrique;
Tomé-Bermejo, Félix;
Adams, Christopher

Publication type:

Article

Anesthetic Management in Mucopolysaccharidoses.

Published in:

ISRN Anesthesiology, 2013, p. 1, doi. 10.1155/2013/791983

By:

Spinello, Concetta Maria;
Novello, Lorena Maria;
Pitino, Sofia;
Raiti, Claudia;
Murabito, Paolo;
Stimoli, Fortunato;
Astuto, Marinella

Publication type:

Article

Novel Therapies.

Published in:: Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 2, p. 57
Publication type:: Article

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.

Published in:: Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 28
Publication type:: Article

Improvement in the range of joint motion in seven patients with mucopolysaccharidosis type II during experimental gene expression-targeted isoflavone therapy (GET IT).

Published in:: Current Medical Literature: Lysosomal Storage Disease, 2012, v. 10, n. 1, p. 15
Publication type:: Article

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

Published in:: Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 78
Publication type:: Article

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.

Published in:: Current Medical Literature: Lysosomal Storage Disease, 2008, v. 8, n. 2, p. 68
Publication type:: Article

ENZYME REPLACEMENT THERAPY FOR MUCOPOLYSACCHARIDOSES.

Published in:

Current Medical Literature: Lysosomal Storage Disease, 2004, v. 4, n. 2, p. 29

By:

Beck, Michael

Publication type:

Article

Mucopolysaccharidosis VI, a Case Report.

Published in:

Journal of Guilan University of Medical Sciences, 2014, v. 22, n. 89, p. 83

By:

Koohmanaee SH.;
Taghavi Z.

Publication type:

Article

Current and potential therapeutic strategies for mucopolysaccharidoses.

Published in:

Journal of Clinical Pharmacy & Therapeutics, 2014, v. 39, n. 3, p. 215, doi. 10.1111/jcpt.12136

By:

Noh, H.;
Lee, J. I.

Publication type:

Article

Elosulfase alfa (BMN 110) for the treatment of mucopolysaccharidosis IVA (Morquio A Syndrome).

Published in:

Expert Review of Clinical Pharmacology, 2016, v. 9, n. 12, p. 1521, doi. 10.1080/17512433.2017.1260000

By:

Hendriksz, Christian J.

Publication type:

Article