Enzyme replacement therapy in adult patients with type I Gaucher disease
Enzyme replacement therapy (ERT) is the standard for the treatment of Gaucher disease (GD). A lifelong intravenous administration of a recombinant analogue of human glucocerebrosidase compensates for the functional deficiency of its own enzyme. The use of ERT has changed the clinical phenotype of GD...
| Main Authors: | , |
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| Format: | Article |
| Language: | Russian |
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"Consilium Medicum" Publishing house
2019-07-01
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| Series: | Терапевтический архив |
| Subjects: | |
| Online Access: | https://ter-arkhiv.ru/0040-3660/article/view/33661 |
| _version_ | 1818684440945098752 |
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| author | R V Ponomarev E A Lukina |
| author_facet | R V Ponomarev E A Lukina |
| author_sort | R V Ponomarev |
| collection | DOAJ |
| description | Enzyme replacement therapy (ERT) is the standard for the treatment of Gaucher disease (GD). A lifelong intravenous administration of a recombinant analogue of human glucocerebrosidase compensates for the functional deficiency of its own enzyme. The use of ERT has changed the clinical phenotype of GD, a severe progressive disease has been turned into the status of an asymptomatic metabolic defect. At the same time, a reduced dosing ERT regimen applied in Gaucher patients who had achieved therapeutic goals has not yet been developed. |
| first_indexed | 2024-12-17T10:50:40Z |
| format | Article |
| id | doaj.art-d5508a293cb94301bad579fea5f5467e |
| institution | Directory Open Access Journal |
| issn | 0040-3660 2309-5342 |
| language | Russian |
| last_indexed | 2024-12-17T10:50:40Z |
| publishDate | 2019-07-01 |
| publisher | "Consilium Medicum" Publishing house |
| record_format | Article |
| series | Терапевтический архив |
| spelling | doaj.art-d5508a293cb94301bad579fea5f5467e2022-12-21T21:52:00Zrus"Consilium Medicum" Publishing houseТерапевтический архив0040-36602309-53422019-07-0191712713110.26442/00403660.2019.07.00032730389Enzyme replacement therapy in adult patients with type I Gaucher diseaseR V Ponomarev0E A Lukina1National Research Center for HematologyNational Research Center for HematologyEnzyme replacement therapy (ERT) is the standard for the treatment of Gaucher disease (GD). A lifelong intravenous administration of a recombinant analogue of human glucocerebrosidase compensates for the functional deficiency of its own enzyme. The use of ERT has changed the clinical phenotype of GD, a severe progressive disease has been turned into the status of an asymptomatic metabolic defect. At the same time, a reduced dosing ERT regimen applied in Gaucher patients who had achieved therapeutic goals has not yet been developed.https://ter-arkhiv.ru/0040-3660/article/view/33661gaucher diseaseenzyme replacement therapylow doses regimen |
| spellingShingle | R V Ponomarev E A Lukina Enzyme replacement therapy in adult patients with type I Gaucher disease Терапевтический архив gaucher disease enzyme replacement therapy low doses regimen |
| title | Enzyme replacement therapy in adult patients with type I Gaucher disease |
| title_full | Enzyme replacement therapy in adult patients with type I Gaucher disease |
| title_fullStr | Enzyme replacement therapy in adult patients with type I Gaucher disease |
| title_full_unstemmed | Enzyme replacement therapy in adult patients with type I Gaucher disease |
| title_short | Enzyme replacement therapy in adult patients with type I Gaucher disease |
| title_sort | enzyme replacement therapy in adult patients with type i gaucher disease |
| topic | gaucher disease enzyme replacement therapy low doses regimen |
| url | https://ter-arkhiv.ru/0040-3660/article/view/33661 |
| work_keys_str_mv | AT rvponomarev enzymereplacementtherapyinadultpatientswithtypeigaucherdisease AT ealukina enzymereplacementtherapyinadultpatientswithtypeigaucherdisease |