Glucocerebrosidase level in the cerebrospinal fluid during enzyme replacement therapy – unsuccessful treatment of the neurological abnormality in type 2 Gaucher disease
| Autor: | Makoto Migita, Juri Fujimura, Hisamitsu Hamada, Takashi Shimada, Atsushi Watanabe, Yoshitaka Fukunaga |
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| Rok vydání: | 2003 |
| Předmět: |
medicine.medical_specialty
business.industry Enzyme replacement therapy Disease medicine.disease Gastroenterology Central nervous system disease Glucocerebrosidase activity Endocrinology Cerebrospinal fluid Neurological abnormality Internal medicine Pediatrics Perinatology and Child Health medicine business Glucocerebrosidase |
| Zdroj: | European Journal of Pediatrics. 162:524-525 |
| ISSN: | 1432-1076 0340-6199 |
| Popis: | A girl with type 2 Gaucher disease was treated with macrophage targeted glucocerebrosidase (Cerezyme), and even with high dose Cerezyme therapy, the glucocerebrosidase activity in the CSF was too low to improve neurological symptoms. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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