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A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?
Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-orphan disease, which until 15 years ago had limited treatment options. Eculizumab, a monoclonal antibody that inhibits C5 in the terminal complement cascade, has revolutionised treatment for this disease, near normalising life expectancy and im...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10032435/ https://www.ncbi.nlm.nih.gov/pubmed/37180495 http://dx.doi.org/10.1177/2633004020959349 |