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

Orphanet Journal of Rare Diseases

Volumn 8, Issue 1, 2013, Pages

  Tilson, Hugh ^a   Primatesta, Paola ^b   Kim, Dennis ^c   Rauer, Barbara ^b   Hawkins, Philip N ^d   Hoffman, Hal M ^e   Kuemmerle Deschner, Jasmin ^f   Van Der Poll, Tom ^g   Walker, Ulrich A ^h  

^a IL   (United States)

^b CAPS Registry Steering Committee   (United States)

^c NOVARTIS PHARMA AG   (Switzerland)

^d ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^g UNIVERSITY OF AMSTERDAM   (Netherlands)

^h Rheumatologische Universitatsklinik und Poliklinik   (Switzerland)

Author keywords

Cryopyrin associated periodic syndrome; Epidemiology; Observational study; Registry

Indexed keywords

CANAKINUMAB;

ADOLESCENT; ADULT; ARTICLE; CHILD; CINCA SYNDROME; DISEASE REGISTRY; DRUG EFFICACY; DRUG SAFETY; EXPERIMENTAL THERAPY; FEMALE; FOLLOW UP; HUMAN; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; PATIENT MONITORING; PRESCHOOL CHILD; SCHOOL CHILD;

ADOLESCENT; ANTIBODIES, MONOCLONAL; CHILD; CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES; FEMALE; HUMANS; INTERNET; MALE; RARE DISEASES; REGISTRIES;

EID: 84883721856     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-8-139     Document Type: Article

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