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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...

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Detalles Bibliográficos
Autores principales: Griffin, Morag, Kelly, Richard, Pike, Alexandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2020
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