Works matching Enzyme therapy

Results: 5000

Production of Long-Acting CNGRC–CPG2 Fusion Proteins: New Derivatives to Overcome Drug Immunogenicity of Ligand-Directed Enzyme Prodrug Therapy for Targeted Cancer Treatment.

Published in:

Technology in Cancer Research & Treatment, 2021, p. 1, doi. 10.1177/15330338211057371

By:

Al-mansoori, Layla;
Al Qahtani, Alanod D.;
Elsinga, Philip;
Goda, Sayed K.

Publication type:

Article

Exercise, nutrition and enzyme replacement therapy are efficacious in adult Pompe patients: report from EPOC Consortium.

Published in:

European Journal of Translational Myology, 2021, v. 31, n. 2, p. 1, doi. 10.4081/ejtm.2021.9798

By:

Angelini, Corrado

Publication type:

Article

Gut microbiome as a response marker for pancreatic enzyme replacement therapy in a porcine model of exocrine pancreas insufficiency.

Published in:

Microbial Cell Factories, 2020, v. 19, n. 1, p. N.PAG, doi. 10.1186/s12934-020-01482-2

By:

Ritz, Sabrina;
Hahn, Daniela;
Wami, Haleluya T.;
Tegelkamp, Karin;
Dobrindt, Ulrich;
Schnekenburger, Juergen

Publication type:

Article

Enzyme replacement therapy and immunotherapy lead to significant functional improvement in two children with Pompe disease: a case report.

Published in:

2024

By:

Castellar-Leones, Sandra Milena;
Ortiz-Corredor, Fernando;
Manrique-Hernández, Daniel;
Sánchez-Peñarete, Diana;
Ruiz-Ospina, Edicson;
Soto-Peña, Diana;
Correa-Arrieta, Cristian

Publication type:

Case Study

Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia.

Published in:

Journal of Bone & Mineral Research, 2024, v. 39, n. 10, p. 1412, doi. 10.1093/jbmr/zjae128

By:

Dahir, Kathryn M;
Shannon, Amy;
Dunn, Derek;
Voegtli, Walter;
Dong, Qunming;
Hasan, Jawad;
Pradhan, Rajendra;
Pelto, Ryan;
Pan, Wei-Jian

Publication type:

Article

Long- and Short-Term Glucosphingosine (lyso-Gb1) Dynamics in Gaucher Patients Undergoing Enzyme Replacement Therapy.

Published in:

Biomolecules (2218-273X), 2024, v. 14, n. 7, p. 842, doi. 10.3390/biom14070842

By:

Dubiela, Pawel;
Szymanska-Rozek, Paulina;
Hasinski, Piotr;
Lipinski, Patryk;
Kleinotiene, Grazina;
Giersz, Dorota;
Tylki-Szymanska, Anna

Publication type:

Article

Development of the "Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease" Based on 6 Years Treatment Experience in 48 Patients.

Published in:

Journal of Child Neurology, 2021, v. 36, n. 8, p. 635, doi. 10.1177/0883073821989154

By:

Schwering, Christoph;
Kammler, Gertrud;
Wibbeler, Eva;
Christner, Martin;
Knobloch, Johannes K.-M.;
Nickel, Miriam;
Denecke, Jonas;
Baehr, Michael;
Schulz, Angela

Publication type:

Article

Abbreviation of Desensitization Protocol for Pediatric Patients with Lysosomal Storage Diseases Receiving Enzyme Replacement Therapy.

Published in:

International Archives of Allergy & Immunology, 2024, v. 185, n. 12, p. 1226, doi. 10.1159/000539597

By:

Yörüsün, Gökhan;
Yilmaz Topal, Özge;
Şengül Emeksiz, Zeynep;
Küçükçongar Yavaş, Aynur;
Kasapkara, Çiğdem Seher;
Dibek Mısırlıoğlu, Emine

Publication type:

Article

ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS?

Published in:

Gomal Journal of Medical Sciences, 2021, v. 19, n. 2, p. 73, doi. 10.46903/gjms/19.02.999

By:

Hassan, Taimoor;
Xu Huadong

Publication type:

Article

How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases?

Published in:

Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-025-03844-8

By:

Spataro, Federico;
Solimando, Antonio Giovanni;
Desantis, Vanessa;
Vacca, Angelo;
Di Girolamo, Attilio;
Ria, Roberto

Publication type:

Article

Assessment of the function and morphology of the thyroid gland in paediatric patients treated with enzyme replacement therapy due to selected storage diseases - preliminary results of our own research and a review of the literature.

Published in:

Pediatric Endocrinology, Diabetes & Metabolism, 2022, v. 28, n. 2, p. 114, doi. 10.5114/pedm.2022.112860

By:

Furtak, Aleksandra;
Wędrychowicz, Anna;
Roztoczyńska, Dorota;
Januś, Dominika;
Orchel-Szastak, Karolina;
Kwinta, Przemko;
Starzyk, Jerzy B.

Publication type:

Article

Inflammatory cytokine expression in Fabry disease: impact of disease phenotype and alterations under enzyme replacement therapy.

Published in:

Frontiers in Immunology, 2024, p. 01, doi. 10.3389/fimmu.2024.1367252

By:

Yujing Yuan;
Yawen Zhao;
Fan Li;
Chen Ling;
Yuan Wu;
Wei Ma;
Zhaoxia Wang;
Yun Yuan;
Hongjun Hao;
Wei Zhang

Publication type:

Article

Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.

Published in:

Frontiers in Immunology, 2024, p. 1, doi. 10.3389/fimmu.2024.1336599

By:

Hui-An Chen;
Rai-Hseng Hsu;
Ching-Ya Fang;
Desai, Ankit K.;
Ni-Chung Lee;
Wuh-Liang Hwu;
Fuu-Jen Tsai;
Kishnani, Priya S.;
Yin-Hsiu Chien

Publication type:

Article

Complement activation and cellular inflammation in Fabry disease patients despite enzyme replacement therapy.

Published in:

Frontiers in Immunology, 2024, p. 1, doi. 10.3389/fimmu.2024.1307558

By:

Laffer, Björn;
Lenders, Malte;
Ehlers-Jeske, Elvira;
Heidenreich, Karin;
Brand, Eva;
Köhl, Jörg

Publication type:

Article

Immunophenotype associated with high sustained antibody titers against enzyme replacement therapy in infantile-onset Pompe disease.

Published in:

Frontiers in Immunology, 2024, p. 1, doi. 10.3389/fimmu.2023.1301912

By:

Desai, Ankit K.;
Smith, P. Brian;
Yi, John S.;
Rosenberg, Amy S.;
Burt, Trevor D.;
Kishnani, Priya S.

Publication type:

Article

Antibody-Directed Enzyme Prodrug Therapy: A Promising Approach for a Selective Treatment of Cancer Based on Prodrugs and Monoclonal Antibodies.

Published in:

Chemical Biology & Drug Design, 2009, v. 74, n. 3, p. 205, doi. 10.1111/j.1747-0285.2009.00856.x

By:

Tietze, Lutz F.;
Krewer, Birgit

Publication type:

Article

Enzyme replacement therapy improves erythropoiesis and iron dysregulation in Gaucher disease.

Published in:

Annals of Hematology, 2024, v. 103, n. 12, p. 5113, doi. 10.1007/s00277-024-05918-2

By:

Motta, Irene;
Delbini, Paola;
Scaramellini, Natalia;
Ghiandai, Viola;
Duca, Lorena;
Nava, Isabella;
Nascimbeni, Fabio;
Lugari, Simonetta;
Consonni, Dario;
Trombetta, Elena;
Di Stefano, Valeria;
Migone De Amicis, Margherita;
Cassinerio, Elena;
Carubbi, Francesca;
Cappellini, Maria Domenica

Publication type:

Article

Nowe możliwości enzymatycznej terapii zastępczej w chorobie Fabry'ego - pegunigalzydaza alfa.

Published in:

Polish Nephrology & Dialyzotherapy / Nefrologia i Dializoterapia Polska, 2023, v. 27, n. 4, p. 80

By:

Nowicki, Michał

Publication type:

Article

Successful treatment of Gaucher disease type 1 by enzyme replacement therapy over a 10-year duration in a Japanese pediatric patient: A case report.

Published in:

Experimental & Therapeutic Medicine, 2021, v. 21, n. 3, p. N.PAG, doi. 10.3892/etm.2021.9677

By:

Oto, Yuji;
Inoue, Takeshi;
Nagai, So;
Tanaka, Shinichiro;
Itabashi, Hisashi;
Shiraisihi, Masahisa;
Nitta, Akihisa;
Murakami, Nobuyuki;
Ida, Hiroyuki;
Matsubara, Tomoyo

Publication type:

Article

Enzyme replacement therapy for hypophosphatasia—The current paradigm.

Published in:

Clinical Endocrinology, 2024, v. 101, n. 6, p. 593, doi. 10.1111/cen.15063

By:

Schindeler, Aaron;
Ludwig, Karissa;
Munns, Craig F.

Publication type:

Article

Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series.

Published in:

2021

By:

İnci, Aslı;
Okur, İlyas;
Tümer, Leyla;
Biberoğlu, Gürsel;
Öktem, Murat;
Ezgü, Fatih

Publication type:

journal article

Nine healthy deliveries in four pregnant women with enzyme replacement therapy in Gaucher disease.

Published in:

Annals of Medical of Research, 2023, v. 30, n. 2, p. 278, doi. 10.5455/annalsmedres.2023.01.018

By:

Aladag, Hulya;
Aladag, Murat

Publication type:

Article

Role of the Lactide:Glycolide Ratio in PLGA Nanoparticle Stability and Release under Lysosomal Conditions for Enzyme Replacement Therapy of Lysosomal Storage Disorders.

Published in:

Journal of Functional Biomaterials, 2023, v. 14, n. 9, p. 440, doi. 10.3390/jfb14090440

By:

del Moral, Maria;
Loeck, Maximilian;
Muntimadugu, Eameema;
Vives, Guillem;
Pham, Vy;
Pfeifer, Peter;
Battaglia, Giuseppe;
Muro, Silvia

Publication type:

Article

Exocrine Pancreatic Insufficiency Dosing Guidelines for Pancreatic Enzyme Replacement Therapy Vary Widely Across Disease Types.

Published in:

Digestive Diseases & Sciences, 2024, v. 69, n. 2, p. 615, doi. 10.1007/s10620-023-08184-w

By:

Lewis, Dana M.;
Rieke, Jorden G.;
Almusaylim, Khaleal;
Kanchibhatla, Anuhya;
Blanchette, Julia E.;
Lewis, Claudia

Publication type:

Article

Pancreatic Enzyme Replacement Therapy in Patients with Non-pancreatic Digestive Conditions: A Nationwide Claims Analysis.

Published in:

Digestive Diseases & Sciences, 2023, v. 68, n. 5, p. 1754, doi. 10.1007/s10620-022-07750-y

By:

Phillips, Anna Evans;
Tang, Gong;
Wang, Xianling;
Forsmark, Christopher E.;
Yadav, Dhiraj

Publication type:

Article

Two successful pregnancies in a woman with Fabry continuing enzyme replacement therapy.

Published in:

Therapeutic Apheresis & Dialysis, 2024, v. 28, n. 2, p. 329, doi. 10.1111/1744-9987.14081

By:

Paydas, Saime;
Akcabay, Cigdem

Publication type:

Article

Pulmonary manifestations and the effectiveness of enzyme replacement therapy in Fabry Disease with the p. Arg227Ter (p.R227*) mutation.

Published in:

Molecular Genetics & Genomic Medicine, 2022, v. 10, n. 5, p. 1, doi. 10.1002/mgg3.1915

By:

Pietilä‐Effati, Päivi;
Söderström, Johan;
Saarinen, Jukka T.;
Löyttyniemi, Eliisa;
Kantola, Ilkka

Publication type:

Article

Hypersensitivity reaction during enzyme replacement therapy in lysosomal storage disorders. A systematic review of desensitization strategies.

Published in:

Pediatric Allergy & Immunology, 2023, v. 34, n. 6, p. 1, doi. 10.1111/pai.13981

By:

Spataro, Federico;
Carlucci, Palma;
Loverre, Teresa;
Macchia, Luigi;
Di Bona, Danilo

Publication type:

Article

Development of Lanzyme as the Potential Enzyme Replacement Therapy Drug for Fabry Disease.

Published in:

Biomolecules (2218-273X), 2023, v. 13, n. 1, p. 53, doi. 10.3390/biom13010053

By:

Deng, Mulan;
Zhou, Hongyu;
Liang, Zhicheng;
Li, Zhaoyang;
Wang, Yanping;
Guo, Wanyi;
Zhao, April Yuanyi;
Li, Fanghong;
Mu, Yunping;
Zhao, Allan Zijian

Publication type:

Article

Neuropsychiatric Symptoms and Their Association With Sex, Age, and Enzyme Replacement Therapy in Fabry Disease: A Systematic Review.

Published in:

Frontiers in Psychiatry, 2022, v. 13, p. 1, doi. 10.3389/fpsyt.2022.829128

By:

Mroczek, Magdalena;
Maniscalco, Ignazio;
Sendel, Manon;
Baron, Ralf;
Seifritz, Erich;
Nowak, Albina

Publication type:

Article

Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID‐19 pandemic.

Published in:

European Journal of Neurology, 2022, v. 29, n. 4, p. 1181, doi. 10.1111/ene.15222

By:

Tard, Céline;
Salort‐Campana, Emmanuelle;
Michaud, Maud;
Spinazzi, Marco;
Nadaj Pakleza, Aleksandra;
Durr, Hélène;
Bouhour, Françoise;
Lefeuvre, Claire;
Thomas, Romain;
Arrassi, Azzeddine;
Taouagh, Nadjib;
Solé, Guilhem;
Laforêt, Pascal;
P, Laforêt;
D, Orlikowski;
G, Bassez;
D, Germain;
JB, Noury;
F, Zagnoli;
C, Tard

Publication type:

Article

Musculoskeletal Comorbidities and Quality of Life in ENPP1‐Deficient Adults and the Response of Enthesopathy to Enzyme Replacement Therapy in Murine Models.

Published in:

Journal of Bone & Mineral Research, 2022, v. 37, n. 3, p. 494, doi. 10.1002/jbmr.4487

By:

Ferreira, Carlos R.;
Ansh, Anenya Jai;
Nester, Catherine;
O'Brien, Christine;
Stabach, Paul R.;
Murtada, Sae‐Il;
Lester, Ethan R.;
Khursigara, Gus;
Molloy, Liz;
Carpenter, Thomas O.;
Braddock, Demetrios T.

Publication type:

Article

Home Enzyme Replacement Therapy in Gaucher Disease: A Review.

Published in:

Journal of Clinical Medicine, 2025, v. 14, n. 3, p. 842, doi. 10.3390/jcm14030842

By:

Kieć-Wilk, Beata;
Guijt, Paul;
Dan, Michaela;
Abdelwahab, Magy;
Revel-Vilk, Shoshana;
Serratrice, Christine

Publication type:

Article

A Feasibility Open-Labeled Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy.

Published in:

Journal of Clinical Medicine, 2024, v. 13, n. 11, p. 3325, doi. 10.3390/jcm13113325

By:

Hurvitz, Noa;
Dinur, Tama;
Revel-Vilk, Shoshana;
Agus, Samuel;
Berg, Marc;
Zimran, Ari;
Ilan, Yaron

Publication type:

Article

The Bone Biomarker of Quantitative Chemical Shift Imaging in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long-Term Enzyme Replacement Therapy.

Published in:

Journal of Clinical Medicine, 2023, v. 12, n. 6, p. 2220, doi. 10.3390/jcm12062220

By:

Zimran, Ari;
Szer, Jeff;
Becker-Cohen, Michal;
Jens, Sjoerd;
Cozma, Claudia;
Revel-Vilk, Shoshana

Publication type:

Article

Downregulation of Mannose-6-Phosphate Receptors in Fabry Disease Cardiomyopathy: A Potential Target for Enzyme Therapy Enhancement.

Published in:

Journal of Clinical Medicine, 2022, v. 11, n. 18, p. N.PAG, doi. 10.3390/jcm11185440

By:

Frustaci, Andrea;
Verardo, Romina;
Scialla, Rossella;
Bagnato, Giulia;
Verardo, Margherita;
Alfarano, Maria;
Russo, Matteo A.

Publication type:

Article

Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).

Published in:

Journal of Clinical Medicine, 2022, v. 11, n. 17, p. 5158, doi. 10.3390/jcm11175158

By:

Hughes, Derralynn A.;
Deegan, Patrick;
Giraldo, Pilar;
Göker-Alpan, Özlem;
Lau, Heather;
Lukina, Elena;
Revel-Vilk, Shoshana;
Scarpa, Maurizio;
Botha, Jaco;
Gadir, Noga;
Zimran, Ari

Publication type:

Article

Divergent Impact of Enzyme Replacement Therapy on Human Cardiomyocytes and Enterocytes Affected by Fabry Disease: Correlation with Mannose-6-phosphate Receptor Expression.

Published in:

Journal of Clinical Medicine, 2022, v. 11, n. 5, p. 1344, doi. 10.3390/jcm11051344

By:

Frustaci, Andrea;
Najafian, Behzad;
Donato, Giuseppe;
Verardo, Romina;
Chimenti, Cristina;
Sansone, Luigi;
Belli, Manuel;
Vernucci, Enza;
Russo, Matteo Antonio

Publication type:

Article

A Systematic Review and Meta-Analysis of Enzyme Replacement Therapy in Late-Onset Pompe Disease.

Published in:

Journal of Clinical Medicine, 2021, v. 10, n. 21, p. 4828, doi. 10.3390/jcm10214828

By:

Dornelles, Alícia Dorneles;
Junges, Ana Paula Pedroso;
Pereira, Tiago Veiga;
Krug, Bárbara Corrêa;
Gonçalves, Candice Beatriz Treter;
Llerena Jr., Juan Clinton;
Kishnani, Priya Sunil;
de Oliveira Jr., Haliton Alves;
Schwartz, Ida Vanessa Doederlein

Publication type:

Article

Individualized Assessment of Exercise Capacity in Response to Acute and Long-Term Enzyme Replacement Therapy in Pediatric Pompe Disease.

Published in:

Journal of Personalized Medicine, 2021, v. 11, n. 11, p. 1105, doi. 10.3390/jpm11111105

By:

Bar-Yoseph, Ronen;
Tal, Galit;
Dumin, Elena;
Hanna, Moneera;
Mainzer, Gur;
Zucker-Toledano, Merav;
Shallufi, George;
Jahshan, Mira;
Mandel, Hanna;
Bentur, Lea

Publication type:

Article

Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease.

Published in:

2023

By:

Fiege, Lina;
Duran, Ibrahim;
Marquardt, Thorsten

Publication type:

Case Study

Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.

Published in:

2021

By:

Potter, Jane E.;
Petts, Gemma;
Ghosh, Arunabha;
White, Fiona J.;
Kinsella, Jane L.;
Hughes, Stephen;
Roberts, Jane;
Hodgkinson, Adam;
Brammeier, Kathryn;
Church, Heather;
Merrigan, Christine;
Hughes, Joanne;
Evans, Pamela;
Campbell, Helen;
Bonney, Denise;
Newman, William G.;
Bigger, Brian W.;
Broomfield, Alexander;
Jones, Simon A.;
Wynn, Robert F.

Publication type:

journal article

Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series.

Published in:

2021

By:

Kılavuz, Sebile;
Basaran, Sibel;
Kor, Deniz;
Bulut, Fatma Derya;
Erdem, Sevcan;
Ballı, Hüseyin Tuğsan;
Dağkıran, Muhammed;
Bisgin, Atil;
Mungan, Halise Neslihan Önenli

Publication type:

journal article

Changes in PCSK 9 and apolipoprotein B100 in Niemann-Pick disease after enzyme replacement therapy with olipudase alfa.

Published in:

2021

By:

Garside, Bethanie;
Ho, Jan Hoong;
Kwok, See;
Liu, Yifen;
Dhage, Shaishav;
Donn, Rachelle;
Iqbal, Zohaib;
Jones, Simon A.;
Soran, Handrean

Publication type:

journal article

Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study.

Published in:

2020

By:

Ficicioglu, Can;
Matalon, Dena R.;
Luongo, Nicole;
Menello, Caitlin;
Kornafel, Tracy;
Degnan, Andrew J.

Publication type:

journal article

Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-Year Clinical Outcome Study of the First Taiwanese Case.

Published in:

Diagnostics (2075-4418), 2025, v. 15, n. 4, p. 464, doi. 10.3390/diagnostics15040464

By:

Lee, Chung-Lin;
Chuang, Chih-Kuang;
Chiu, Huei-Ching;
Chang, Ya-Hui;
Tu, Yuan-Rong;
Lo, Yun-Ting;
Lin, Hsiang-Yu;
Lin, Shuan-Pei

Publication type:

Article

Aortic Root Dilatation in Taiwanese Patients with Mucopolysaccharidoses and the Long-Term Effects of Enzyme Replacement Therapy.

Published in:

Diagnostics (2075-4418), 2021, v. 11, n. 1, p. 16, doi. 10.3390/diagnostics11010016

By:

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

Publication type:

Article

Cost-effectiveness analysis of enzyme replacement therapy for the treatment of Chinese patients with fabry disease: a Markov model.

Published in:

Frontiers in Pharmacology, 2025, p. 1, doi. 10.3389/fphar.2025.1546018

By:

Huang, Yueyang;
Yuan, Hongmei;
Huang, Zhe

Publication type:

Article

Clinical Profiles of Children with Hypophosphatasia prior to Treatment with Enzyme Replacement Therapy: An Observational Analysis from the Global HPP Registry.

Published in:

Hormone Research in Paediatrics, 2024, v. 97, n. 3, p. 233, doi. 10.1159/000531865

By:

Martos-Moreno, Gabriel Ángel;
Rockman-Greenberg, Cheryl;
Ozono, Keiichi;
Petryk, Anna;
Kishnani, Priya S.;
Dahir, Kathryn M.;
Seefried, Lothar;
Fang, Shona;
Högler, Wolfgang;
Linglart, Agnès

Publication type:

Article

Lethal Encephalopathy in an Infant with Hypophosphatasia despite Enzyme Replacement Therapy.

Published in:

Hormone Research in Paediatrics, 2021, v. 94, n. 9/10, p. 390, doi. 10.1159/000520341

By:

Raimann, Adalbert;
Haberler, Christine;
Patsch, Janina;
Ertl, Diana-Alexandra;
Sadeghi, Kambis;
Freilinger, Michael;
Lang, Susanna;
Schmook, Maria;
Plecko, Barbara;
Haeusler, Gabriele

Publication type:

Article