Results 1 to 10 of about 496,759 (383)

Enzyme replacement therapy: efficacy and limitations [PDF]

Italian Journal of Pediatrics, 2018
Enzyme replacement therapy (ERT) is available for mucopolysaccharidosis (MPS) I, MPS II, MPS VI, and MPS IVA. The efficacy of ERT has been evaluated in clinical trials and in many post-marketing studies with a long-term follow-up for MPS I, MPS II, and MPS VI.
Daniela Concolino, Rossella Parini, Federica Deodato   +2 more
doaj   +6 more sources

Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors [PDF]

PLoS ONE, 2017
Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. Identification of factors that predict disease progression is needed to refine guidelines on initiation and cessation of enzyme replacement therapy.
Arends, M, Biegstraaten, M, Elliott, PM, Hollak, CEM, Hughes, DA, Mehta, A, Oder, D, van Kuilenburg, ABP, Vaz, FM, Wanner, C, Watkinson, OT   +10 more
core   +4 more sources

Enzyme replacement therapies: what is the best option? [PDF]

BioImpacts, 2018
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT), several limitations such as the high-cost of the treatment and various inadvertent side effects including the occurrence of an immunological response against the infused enzyme and development of resistance to enzymes persist.
Azam Safary, Mostafa Akbarzadeh Khiavi, Rahimeh Mousavi, Jaleh Barar, Mohammad A Rafi   +4 more
openaire   +7 more sources

Erythrocytes as Carriers of Therapeutic Enzymes. [PDF]

, 2020
Therapeutic enzymes are administered for the treatment of a wide variety of diseases. They exert their effects through binding with a high affinity and specificity to disease-causing substrates to catalyze their conversion to a non-noxious product, to ...
Bax, BE
core   +1 more source

Enzyme Replacement Therapy In Hypophosphatasia

Journal of the College of Physicians and Surgeons Pakistan, 2018
Hypophosphatasia (HPP) is associated with significant morbidity and mortality in pediatric patients. The disease also imposes a high disease-burden in adult-onset HPP. Asfotase alfa (AA) is the first-in-class, bone-targeted, enzyme- replacement therapy designated to reverse the skeletal mineralisation defects in HPP.
Sevim Ünal, S. Ahmet Uçaktürk, Eda Mengen, Deniz Gönülal, Selin Elmaogullari   +4 more
openaire   +5 more sources

A Distinct Urinary Biomarker Pattern Characteristic of Female Fabry Patients That Mirrors Response to Enzyme Replacement Therapy [PDF]

, 2011
Female patients affected by Fabry disease, an X-linked lysosomal storage disorder, exhibit a wide spectrum of symptoms, which renders diagnosis, and treatment decisions challenging.
A Mehta, A Reiner, A Rolfs, AD Kistler, Alexander Scherl, Andreas D. Kistler, Andreas L. Serra, B Sachdev, C Auray-Blais, C Chimenti, C Delles, C Whybra, Christoph Wanner, CM Eng, D Theodorescu, D Theodorescu, David G. Warnock, Derralynn A. Hughes, DF Moore, DM Good, E Paschke, E Schiffer, E Young, Frank Breunig, GE Linthorst, H Mischak, H Mischak, Harald Mischak, Iris Schrijver, J Andrade, J DeLeo, J Jantos-Siwy, J Metzger, JJ Coon, Justyna Siwy, K Rossing, L Vojtova, M Haubitz, M Merta, M Tanaka, MV Baptista, N Guffon, N Neuhoff, P Gaspar, P Kotanko, P Vylet'al, P Zurbig, PA Rozenfeld, PD Whitfield, Praveen Jeevaratnam, R Brouns, R Schiffmann, RJ Desnick, RP El Dib, Rudolf P. Wüthrich, S Decramer, S Nakao, S Wittke, T Kitagawa, V Thongboonkerd, WB Dobyns, William Mullen, WR Wilcox   +62 more
core   +15 more sources

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy. [PDF]

, 2016
BackgroundFollowing a drug manufacturing process change, safety/efficacy of agalsidase alfa were evaluated in enzyme replacement therapy (ERT)-naïve children with Fabry disease.MethodsIn an open-label, multicenter, Phase II study (HGT-REP-084; Shire), 14
Chang, Peter, Goker-Alpan, Ozlem, Longo, Nicola, McDonald, Marie, Pano, Arian, Schiffmann, Raphael, Shankar, Suma P, Shen, Yinghua   +7 more
core   +2 more sources

Enzyme replacement therapy for Gaucher disease [PDF]

Blood, 1991
Four patients with moderately severe type I Gaucher disease were treated with commercially available mannose terminated glucocerebrosidase (Ceredase; Genzyme, Boston, MA) for up to 13 months. The enzyme was administered at the rate of three to four times weekly at one fourth the total recommended dosage, greatly decreasing the cost.
P. Garver, Andrea C. Kay, Ernest Beutler, Arthur Dawson, D. Thurston, Alan Saven, Barry E. Rosenbloom   +6 more
openaire   +4 more sources

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease [PDF]

, 2014
Background: Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease.
Hakkaart-van Roijen, L. (Leona), Hoogenboom-Plug, I (Iris), Kanters, T.A. (Tim), Ploeg, A.T. (Ans) van der, Redekop, W.K. (Ken), Rutten-van Mölken, M.P.M.H. (Maureen)   +5 more
core   +2 more sources

Engineering of GlcNAc-1-phosphotransferase for production of highly phosphorylated lysosomal enzymes for enzyme replacement therapy [PDF]

, 2017
Several lysosomal enzymes currently used for enzyme replacement therapy in patients with lysosomal storage diseases contain very low levels of mannose 6-phosphate, limiting their uptake via mannose 6-phosphate receptors on the surface of the deficient ...
Doray, Balraj, Kornfeld, Stuart, Lee, Wang-Sik, Liu, Lin   +3 more
core   +3 more sources