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Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis
Abstract Background Lymphangioleiomyomatosis is a rare disease caused by unregulated activation of mammalian target of rapamycin (mTOR) signalling pathway. Sirolimus showed efficacy in a phase 3 trial of patients with lymphangioleiomyomatosis, but the optimal dose remains unclear. Methods We investi...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BMC
2018-11-01
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| Colecção: | Orphanet Journal of Rare Diseases |
| Assuntos: | |
| Acesso em linha: | http://link.springer.com/article/10.1186/s13023-018-0946-8 |
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