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Positive effect of erythrocyte-delivered dexamethasone in ataxia-telangiectasia
OBJECTIVE: Ataxia-telangiectasia (AT) is a rare, devastating neurodegenerative disease presenting with early-onset ataxia, oculocutaneous telangiectasia, immunodeficiency, radiosensitivity, and proneness to cancer. In a previous phase 2 study, we showed that 6 monthly infusions of autologous erythro...
Autores principales: | Leuzzi, Vincenzo, Micheli, Roberto, D'Agnano, Daniela, Molinaro, Anna, Venturi, Tullia, Plebani, Alessandro, Soresina, Annarosa, Marini, Mirella, Ferremi Leali, Pierino, Quinti, Isabella, Pietrogrande, Maria C., Finocchi, Andrea, Fazzi, Elisa, Chessa, Luciana, Magnani, Mauro |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Lippincott Williams & Wilkins
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4396528/ https://www.ncbi.nlm.nih.gov/pubmed/25884015 http://dx.doi.org/10.1212/NXI.0000000000000098 |
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