Title |
Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry
|
---|---|
Published in |
Orphanet Journal of Rare Diseases, September 2013
|
DOI | 10.1186/1750-1172-8-139 |
Pubmed ID | |
Authors |
Hugh Tilson, Paola Primatesta, Dennis Kim, Barbara Rauer, Philip N Hawkins, Hal M Hoffman, Jasmin Kuemmerle-Deschner, Tom van der Poll, Ulrich A Walker |
Abstract |
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 antibody directed against IL-1 beta and approved for CAPS patients but requires post-approval monitoring due to low and short exposures during the licensing process. Creative approaches to observational methodology are needed, harnessing novel registry strategies to ensure Health Care Provider reporting and patient monitoring. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Mexico | 1 | 50% |
Unknown | 1 | 50% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 1 | 50% |
Scientists | 1 | 50% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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United Kingdom | 1 | 3% |
Italy | 1 | 3% |
Unknown | 37 | 95% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 7 | 18% |
Researcher | 6 | 15% |
Student > Bachelor | 4 | 10% |
Other | 3 | 8% |
Professor > Associate Professor | 3 | 8% |
Other | 10 | 26% |
Unknown | 6 | 15% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 14 | 36% |
Biochemistry, Genetics and Molecular Biology | 5 | 13% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 5% |
Agricultural and Biological Sciences | 2 | 5% |
Nursing and Health Professions | 2 | 5% |
Other | 7 | 18% |
Unknown | 7 | 18% |