Works matching DE "GAUCHER'S disease treatment"

Results: 115

Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study.

Published in:

Annals of Clinical & Translational Neurology, 2016, v. 3, n. 3, p. 200, doi. 10.1002/acn3.292

By:

Narita, Aya;
Shirai, Kentarou;
Itamura, Shinji;
Matsuda, Atsue;
Ishihara, Akiko;
Matsushita, Kumi;
Fukuda, Chisako;
Kubota, Norika;
Takayama, Rumiko;
Shigematsu, Hideo;
Hayashi, Anri;
Kumada, Tomohiro;
Yuge, Kotaro;
Watanabe, Yoriko;
Kosugi, Saori;
Nishida, Hiroshi;
Kimura, Yukiko;
Endo, Yusuke;
Higaki, Katsumi;
Nanba, Eiji

Publication type:

Article

β-Glycosphingolipids as Mediators of Both Inflammation and Immune Tolerance: A Manifestation of Randomness in Biological Systems.

Published in:

Frontiers in Immunology, 2019, p. N.PAG, doi. 10.3389/fimmu.2019.01143

By:

Ilan, Yaron

Publication type:

Article

Successful mobilization with plerixafor and autologous hematopoietic SCT in a patient with refractory Hodgkin's lymphoma and Gaucher disease.

Published in:

2011

By:

Symeonidis, A.;
Liga, M.;
Triantafyllou, E.;
Kouraklis, A.;
Tiniakou, M.;
Karakantza, M.;
Spyridonidis, A.;
Zoumbos, N.

Publication type:

Letter

Glucocerebrosidase level in the cerebrospinal fluid during enzyme replacement therapy – unsuccessful treatment of the neurological abnormality in type 2 Gaucher disease.

Published in:

European Journal of Pediatrics, 2003, v. 162, n. 7/8, p. 524, doi. 10.1007/s00431-001-0859-7

By:

Migita, Makoto;
Hamada, Hisamitsu;
Fujimura, Juri;
Watanabe, Atsushi;
Shimada, Takashi;
Fukunaga, Yoshitaka

Publication type:

Article

Improvement of neurological symptoms by enzyme replacement therapy for Gaucher disease type IIIb.

Published in:

2001

By:

Aoki, Minako;
Takahashi, Yukitoshi;
Miwa, Yuka;
Iida, Satoko;
Sukegawa, Kazuko;
Horai, Toshio;
Orii, Tadao;
Kondo, Naomi;
Aoki, M;
Takahashi, Y;
Miwa, Y;
Iida, S;
Sukegawa, K;
Horai, T;
Orii, T;
Kondo, N

Publication type:

Case Study

Allogeneic Hematopoietic Stem Cell Transplantation for Treatment of Gaucher Disease.

Published in:

Pediatric Hematology & Oncology, 2013, v. 30, n. 5, p. 459, doi. 10.3109/08880018.2013.793757

By:

Machaczka, Maciej

Publication type:

Article

Response to the Letter: Allogeneic Hematopoietic Stem Cell Transplantation for Treatment of Gaucher Disease.

Published in:

Pediatric Hematology & Oncology, 2013, v. 30, n. 5, p. 462, doi. 10.3109/08880018.2013.793758

By:

Ito, Sawa;
Barrett, A. John

Publication type:

Article

QUALITY OF LIFE OF PATIENTS WITH GAUCHER DISEASE.

Published in:

Journal of Nursing UFPE / Revista de Enfermagem UFPE, 2017, v. 11, n. 11, p. 4282, doi. 10.5205/reuol.23542-49901-1-ED.1111201702

By:

de Oliveira Freitas, Saulo Emanoel;
Correia Ferreira, Themístoclys Thesko;
de Souza Costa, Bruna Gabrielle;
Magalhães Soares, Rafaela;
Cruz de Lucena, Nahãmi;
de Barros Correia, Nancy

Publication type:

Article

TYPE 2 GAUCHER DISEASE: ONSET AND EVOLUTION - CASE REPORT.

Published in:

Romanian Journal of Pediatrics / Revista Romana de Pediatrie, 2015, v. 64, n. 3, p. 293, doi. 10.37897/rjp.2015.3.12

By:

Streanga, Violeta;
Jitareanu, Cristina;
Ciomaga, Irina M.;
Mihaila, Doina;
Nistor, Nicolai

Publication type:

Article

RIPK3 as a potential therapeutic target for Gaucher's disease.

Published in:

Nature Medicine, 2014, v. 20, n. 2, p. 204, doi. 10.1038/nm.3449

By:

Vitner, Einat B;
Salomon, Ran;
Farfel-Becker, Tamar;
Meshcheriakova, Anna;
Ali, Mohammad;
Klein, Andrés D;
Platt, Frances M;
Cox, Timothy M;
Futerman, Anthony H

Publication type:

Article

The natural history of Gaucher disease type 1 in 31 patients over a median of 15 years: a retrospective study.

Published in:

Internal Medicine Journal, 2024, v. 54, n. 10, p. 1661, doi. 10.1111/imj.16503

By:

Piran, Siavash;
Nimmo, Graeme A. M.;
Chaboureau, Amélie;
Mohankumar, Rakesh;
Kalia, Lorraine V.;
Amato, Dominick

Publication type:

Article

IMMUNOGLOBULIN HEAVY CHAIN GENE REARRANGEMENTS IN PATIENTS WITH GAUCHER DISEASE.

Published in:

Journal of Medical Biochemistry, 2018, v. 37, n. 3, p. 307, doi. 10.1515/jomb-2017-0061

By:

Rodić, Predrag;
Lakočevip, Milan;
Pavlović, Šonja;
Đurašević, Teodora Karan;
Kostić, Tatjana;
Vuković, Nada Suvajdžić;
Šumarac, Zorica;
Petakov, Milan;
Janić, Dragana

Publication type:

Article

Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease.

Published in:

PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0128986

By:

Abbas, Richat;
Park, Glen;
Damle, Bharat;
Chertkoff, Raul;
Alon, Sari

Publication type:

Article

Comparative Therapeutic Effects of Velaglucerase Alfa and Imiglucerase in a Gaucher Disease Mouse Model.

Published in:

PLoS ONE, 2010, v. 5, n. 5, p. 1, doi. 10.1371/journal.pone.0010750

By:

You-Hai Xu;
Ying Sun;
Barnes, Sonya;
Grabowski, Gregory A.

Publication type:

Article

2012 The year in review.

Published in:

Chemistry & Industry, 2012, v. 76, n. 11, p. 32

By:

O'Driscoll, Cath

Publication type:

Article

Terapia de reemplazo enzimático en una paciente con enfermedad de Gaucher tipo III.

Published in:

Acta Pediatrica de Mexico, 2012, v. 33, n. 1, p. 9

By:

Carbajal-Rodríguez, Luis;
Gómez-González, Fernanda;
Rodríguez-Herrera, Raymundo;
Zarco-Román, Jorge;
Mora-Tiscareño, Antonieta

Publication type:

Article

Gene variants of osteoprotegerin, estrogen-, calcitonin- and vitamin D-receptor genes and serum markers of bone metabolism in patients with Gaucher disease type 1.

Published in:

2018

By:

Zimmermann, Anca;
Popp, Radu A;
Rossmann, Heidi;
Bucerzan, Simona;
Nascu, Ioana;
Leucuta, Daniel;
Weber, Matthias M;
Grigorescu-Sido, Paula

Publication type:

journal article

Profile of eliglustat tartrate in the management of Gaucher disease.

Published in:

2016

By:

Sechi, Annalisa;
Dardis, Andrea;
Bembi, Bruno

Publication type:

journal article

Gaucher disease.

Published in:

International Journal of Child Health & Human Development, 2020, v. 13, n. 2, p. 161

By:

Hershkop, Eliyakim;
Kurolap, Alina;
Feldman, Hagit Baris

Publication type:

Article

Cytokines in Gaucher disease: Role in the pathogenesis of bone and pulmonary disease.

Published in:

Egyptian Journal of Medical Human Genetics, 2015, v. 16, n. 3, p. 207, doi. 10.1016/j.ejmhg.2015.02.001

By:

Tantawy, Azza A. G.

Publication type:

Article

Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.

Published in:

2022

By:

Eskes, Eline C. B.;
Beishuizen, Cathrien R. L.;
Corazolla, Eleonore M.;
van Middelaar, Tessa;
Brands, Marion M. M. G.;
Dekker, Hanka;
van de Mheen, Erica;
Langeveld, Mirjam;
Hollak, Carla E. M.;
Sjouke, Barbara

Publication type:

journal article

Glucocerebrosidase is shaking up the synucleinopathies.

Published in:

Brain: A Journal of Neurology, 2014, v. 137, n. 5, p. 1304, doi. 10.1093/brain/awu002

By:

Siebert, Marina;
Sidransky, Ellen;
Westbroek, Wendy

Publication type:

Article

Contribution of brain inflammation to neuronal cell death in neuronopathic forms of Gaucher's disease.

Published in:

Brain: A Journal of Neurology, 2012, v. 135, n. 6, p. 1724, doi. 10.1093/brain/aws095

By:

Vitner, Einat B.;
Farfel-Becker, Tamar;
Eilam, Raya;
Biton, Inbal;
Futerman, Anthony H.

Publication type:

Article

Velaglucerase alfa.

Published in:: Australian Prescriber, 2012, v. 35, n. 6, p. 210
Publication type:: Article

Target identification: Targeting neuronal loss in Gaucher's disease.

Published in:

Nature Reviews Drug Discovery, 2014, v. 13, n. 3, p. 178, doi. 10.1038/nrd4270

By:

Harrison, Charlotte

Publication type:

Article

Velaglucerase alfa.

Published in:

2010

By:

Aerts, Johannes M. F. G.;
Yasothan, Uma;
Kirkpatrick, Peter

Publication type:

journal article

Lysosomal storage diseases market.

Published in:

Nature Reviews Drug Discovery, 2004, v. 3, n. 1, p. 9, doi. 10.1038/nrd1286

By:

Werber, Yaron

Publication type:

Article

Total synthesis of N -butyl-1-deoxynojirimycin.

Published in:

Journal of Carbohydrate Chemistry, 2016, v. 35, n. 8/9, p. 445, doi. 10.1080/07328303.2017.1330415

By:

Wang, Jiajia;
Zhao, Yunyan;
Zhao, Wei;
Wang, Peng;
Li, Jing

Publication type:

Article

VPRIV.

Published in:: Formulary, 2010, v. 45, n. 4, p. 113
Publication type:: Article

Low-dose high-frequency enzyme replacement therapy for very young children with severe Gaucher...

Published in:

1994

By:

Zimran, A.;
Smith, Jeanette M.

Publication type:

Abstract

Gaucher's Disease: Diagnosis and Treatment.

Published in:: American Family Physician, 1994, v. 49, n. 5, p. 1231
Publication type:: Article

Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.

Published in:

2018

By:

Amato, Dominick;
Patterson, Mary Anne

Publication type:

journal article

Immunogenicity of glycans on biotherapeutic drugs produced in plant expression systems—The taliglucerase alfa story.

Published in:

PLoS ONE, 2017, v. 12, n. 11, p. 1, doi. 10.1371/journal.pone.0186211

By:

Rup, Bonita;
Alon, Sari;
Amit-Cohen, Bat-Chen;
Brill Almon, Einat;
Chertkoff, Raul;
Tekoah, Yoram;
Rudd, Pauline M.

Publication type:

Article

Investigations on therapeutic glucocerebrosidases through paired detection with fluorescent activity-based probes.

Published in:

PLoS ONE, 2017, v. 12, n. 2, p. 1, doi. 10.1371/journal.pone.0170268

By:

Kallemeijn, Wouter W.;
Scheij, Saskia;
Hoogendoorn, Sascha;
Witte, Martin D.;
Herrera Moro Chao, Daniela;
van Roomen, Cindy P. A. A.;
Ottenhoff, Roelof;
Overkleeft, Herman S.;
Boot, Rolf G.;
Aerts, Johannes M. F. G.

Publication type:

Article

Time of Initiating Enzyme Replacement Therapy Affects Immune Abnormalities and Disease Severity in Patients with Gaucher Disease.

Published in:

PLoS ONE, 2016, v. 11, n. 12, p. 1, doi. 10.1371/journal.pone.0168135

By:

Limgala, Renuka Pudi;
Ioanou, Chidima;
Plassmeyer, Matthew;
Ryherd, Mark;
Kozhaya, Lina;
Austin, Lauren;
Abidoglu, Cem;
Unutmaz, Derya;
Alpan, Oral;
Goker-Alpan, Ozlem

Publication type:

Article

Shire's replacement enzymes validate gene activation.

Published in:

Nature Biotechnology, 2010, v. 28, n. 11, p. 1139, doi. 10.1038/nbt1110-1139a

By:

Moran, Nuala

Publication type:

Article

Pfizer stakes a claim in plant cell–made biopharmaceuticals.

Published in:

Nature Biotechnology, 2010, v. 28, n. 2, p. 107, doi. 10.1038/nbt0210-107

By:

Ratner, Mark

Publication type:

Article

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment.

Published in:

Nature Biotechnology, 2008, v. 26, n. 8, p. 901, doi. 10.1038/nbt.1484

By:

Wang, Jinhai;
Lozier, Jay;
Johnson, Gibbes;
Kirshner, Susan;
Verthelyi, Daniela;
Pariser, Anne;
Shores, Elizabeth;
Rosenberg, Amy

Publication type:

Article

EMEA approves OGS drug rejected by FDA.

Published in:

Nature Biotechnology, 2002, v. 20, n. 9, p. 858, doi. 10.1038/nbt0902-858

By:

Mitchell, Peter

Publication type:

Article

Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.

Published in:

Orphanet Journal of Rare Diseases, 2014, v. 9, n. 1, p. 2, doi. 10.1186/1750-1172-9-51

By:

Dussen, Laura van;
Biegstraaten, Marieke;
Hollak, Carla E. M.;
Dijkgraaf, Marcel G. W.

Publication type:

Article

Reducing selection bias in case-control studies from rare disease registries.

Published in:

2011

By:

Cole, J Alexander;
Taylor, John S;
Hangartner, Thomas N;
Weinreb, Neal J;
Mistry, Pramod K;
Khan, Aneal

Publication type:

journal article

Histologic Findings of Femoral Heads From Patients With Gaucher Disease Treated With Enzyme Replacement.

Published in:

American Journal of Clinical Pathology, 2013, v. 140, n. 1, p. 91, doi. 10.1309/AJCPFVSAEGO67NGT

By:

Lebel, Ehud;
Elstein, Deborah;
Peleg, Ariel;
Reinus, Constantine;
Zimran, Ari;
Amir, Gail

Publication type:

Article

Generation of a Chinese Hamster Ovary Cell Line Producing Recombinant Human Glucocerebrosidase.

Published in:

Journal of Biomedicine & Biotechnology, 2012, v. 2012, p. 1, doi. 10.1155/2012/875383

By:

Branco Novo, Juliana;
Morganti, Ligia;
Moro, Ana Maria;
Paes Leme, Adriana Franco;
de Toledo Serrano, Solange Maria;
Raw, Isaias;
Lee Ho, Paulo

Publication type:

Article

A branch retinal artery occlusion in a patient with Gaucher disease.

Published in:

2012

By:

Bruscolini, Alice;
Pirraglia, Maria;
Restivo, Lucia;
Spinucci, Giovanni;
Abbouda, Alessandro

Publication type:

Report

Quality of life assessment in adults with type 1 Gaucher disease.

Published in:

1999

By:

Masek, Bruce J.;
Sims, Katherine B.;
Bove, Catherine M.;
Korson, Mark S.;
Short, Priscilla;
Norman, Dennis K.;
Masek, B J;
Sims, K B;
Bove, C M;
Korson, M S;
Short, P;
Norman, D K

Publication type:

journal article

Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1.

Published in:

Molecular Genetics & Genomic Medicine, 2018, v. 6, n. 1, p. 27, doi. 10.1002/mgg3.339

By:

Cerón‐Rodríguez, Magdalena;
Barajas‐Colón, Edgar;
Ramírez‐Devars, Lyuva;
Gutiérrez‐Camacho, Claudia;
Salgado‐Loza, Juan L.

Publication type:

Article

From the National Institutes of Health.

Published in:

1991

By:

Healy, Bernadine;
Healy, B

Publication type:

journal article

Miglustat: withdrawal by manufacturer.

Published in:: WHO Drug Information, 2008, v. 22, n. 1, p. 21
Publication type:: Article

Rare health conditions 40: Gaucher’s disease, Pompe disease, Ellis-Van Creveld syndrome, and Duchenne muscular dystrophy.

Published in:

British Journal of Healthcare Assistants, 2020, v. 14, n. 10, p. 500, doi. 10.12968/bjha.2020.14.10.500

By:

Barber, Chris

Publication type:

Article

α-Synuclein accumulation and GBA deficiency due to L444P GBA mutation contributes to MPTP-induced parkinsonism.

Published in:

Molecular Neurodegeneration, 2018, v. 12, p. 1, doi. 10.1186/s13024-017-0233-5

By:

Yun, Seung Pil;
Kim, Donghoon;
Kim, Sangjune;
Kim, SangMin;
Karuppagounder, Senthilkumar S.;
Kwon, Seung-Hwan;
Lee, Saebom;
Kam, Tae-In;
Lee, Suhyun;
Ham, Sangwoo;
Park, Jae Hong;
Dawson, Valina L.;
Dawson, Ted M.;
Lee, Yunjong;
Ko, Han Seok

Publication type:

Article