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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3848430/
https://www.ncbi.nlm.nih.gov/pubmed/24016338
http://dx.doi.org/10.1186/1750-1172-8-139

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3848430/
https://www.ncbi.nlm.nih.gov/pubmed/24016338
http://dx.doi.org/10.1186/1750-1172-8-139

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