Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry

BACKGROUND: The Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare hereditary autoinflammatory diseases and encompass Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID). Canakinumab is a monoclonal...

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Detalhes bibliográficos
Main Authors: Tilson, Hugh, Primatesta, Paola, Kim, Dennis, Rauer, Barbara, Hawkins, Philip N, Hoffman, Hal M, Kuemmerle-Deschner, Jasmin, van der Poll, Tom, Walker, Ulrich A
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2013
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3848430/
https://ncbi.nlm.nih.gov/pubmed/24016338
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-139
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