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Evolution of tyrosinemia type 1 disease in patients treated with nitisinone in Spain
Treatment with nitisinone (NTBC) has brought about a drastic improvement in the treatment and prognosis of hereditary tyrosinemia type I (HT1). We conducted a retrospective observational multicentric study in Spanish HT1 patients treated with NTBC to assess clinical and biochemical long-term evoluti...
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| Опубликовано в: : | Medicine (Baltimore) |
|---|---|
| Главные авторы: | , , , , , , , , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Wolters Kluwer Health
2019
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6775438/ https://ncbi.nlm.nih.gov/pubmed/31574857 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1097/MD.0000000000017303 |
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