Works matching DE "MUCOPOLYSACCHARIDOSIS treatment"

Results: 71

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.

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

Thrombocytopenia associated with galsulfase treatment.

Published in:

2011

By:

Doğan, Murat;
Cesur, Yasar;
Peker, Erdal;
Oner, Ahmet F.;
Dogan, Sekibe Zehra

Publication type:

Case Study

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

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

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

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

Laronidase.

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

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

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

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

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

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

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

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

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

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

Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0565-y

By:

Fraldi, Alessandro;
Serafini, Marta;
Sorrentino, Nicolina Cristina;
Gentner, Bernhard;
Aiuti, Alessandro;
Bernardo, Maria Ester

Publication type:

Article

Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0561-2

By:

Barone, Rita;
Pellico, Alessandra;
Pittalà, Annarita;
Gasperini, Serena

Publication type:

Article

The role of imaging in the skeletal involvement of mucopolysaccharidoses.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0556-z

By:

Spina, Vincenzo;
Barbuti, Domenico;
Gaeta, Alberto;
Palmucci, Stefano;
Soscia, Ernesto;
Grimaldi, Marco;
Leone, Antonio;
Manara, Renzo;
Polonara, Gabriele

Publication type:

Article

ENT and mucopolysaccharidoses.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0555-0

By:

Bianchi, Pier Marco;
Gaini, Renato;
Vitale, Silvano

Publication type:

Article

Newborn screening in mucopolysaccharidoses.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0552-3

By:

Donati, Maria Alice;
Pasquini, Elisabetta;
Spada, Marco;
Polo, Giulia;
Burlina, Alberto

Publication type:

Article

Mucopolysaccharidoses: early diagnostic signs in infants and children.

Published in:

Italian Journal of Pediatrics, 2018, v. 44, n. 2, p. N.PAG, doi. 10.1186/s13052-018-0550-5

By:

Galimberti, Cinzia;
Madeo, Annalisa;
Di Rocco, Maja;
Fiumara, Agata

Publication type:

Article

The new frame for Mucopolysaccharidoses.

Published in:

2018

By:

Parini, Rossella;
Biondi, Andrea

Publication type:

Editorial

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

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

Anterior Hypopituitarism and Treatment Response in Hunter Syndrome: A Comparison of Two Patients.

Published in:

2016

By:

Nour, Munier A.;
Luca, Paola;
Stephure, David;
Wei, Xing-Chang;
Khan, Aneal

Publication type:

Case Study

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

Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials.

Published in:

2019

By:

Qi, Yulan;
McKeever, Kathleen;
Taylor, Julie;
Haller, Christine;
Song, Wenjie;
Jones, Simon A.;
Shi, Jack

Publication type:

journal article

Correction to: Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials.

Published in:

2019

By:

Qi, Yulan;
McKeever, Kathleen;
Taylor, Julie;
Haller, Christine;
Song, Wenjie;
Jones, Simon A.;
Shi, Jack

Publication type:

corrected article

Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome.

Published in:

Clinical Pharmacokinetics, 2014, v. 53, n. 12, p. 1137, doi. 10.1007/s40262-014-0173-y

By:

Qi, Yulan;
Musson, Donald;
Schweighardt, Becky;
Tompkins, Troy;
Jesaitis, Lynne;
Shaywitz, Adam;
Yang, Ke;
O'Neill, Charles

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

Quality of life in mucopolysaccharidoses: construction of a specific measure using the focus group technique.

Published in:

BMC Research Notes, 2018, v. 11, p. 1, doi. 10.1186/s13104-018-3157-4

By:

Oliveira, M. R.;
Schwartz, I.;
Costa, L. S.;
Maia, H.;
Ribeiro, M.;
Guerreiro, L. B.;
Acosta, A.;
Rocha, N. S.

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

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

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

Abstract- Poster.

Published in:: 2022
Publication type:: Abstract

Unfavourable outcome after uneventful anaesthesia and surgery in a child with Hurler syndrome.

Published in:

2017

By:

Sinha, Renu;
Kumar, Kanil Ranjith;
Anand, Rahul Kumar;
Ray, Bikash Ranjan

Publication type:

Letter to the Editor

Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.

Published in:

Journal of Molecular Medicine, 2017, v. 95, n. 10, p. 1043, doi. 10.1007/s00109-017-1562-0

By:

Lau, Adeline;
Hemsley, Kim

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