RT Journal Article
T1 Creating an effective clinical registry for rare diseases.
A1 D'Agnolo, Hedwig Ma
A1 Kievit, Wietske
A1 Andrade, Raul J
A1 Karlsen, Tom Hemming
A1 Wedemeyer, Heiner
A1 Drenth, Joost Ph
K1 Clinical registry
K1 database
K1 gastrointestinal disease
K1 liver disease
K1 liver transplantation
K1 practical guide
K1 rare diseases
K1 rare liver disorders
K1 registry design
AB The exposure of clinicians to patients with rare gastrointestinal diseases is limited. This hurts clinical studies, which impedes accumulation of scientific knowledge on the natural disease course, treatment outcomes and prognosis in these patients. An excellent method to detect patterns on an aggregate level that would not be possible to discover in individual cases, is a registry study. This paper aims to describe a template to create a successful international registry for rare diseases. We focus mainly on rare hepatic diseases, but lessons from this paper serve other fields in medicine, as well.
SN 2050-6406
YR 2015
FD 2015-11-13
LK https://hdl.handle.net/10668/26345
UL https://hdl.handle.net/10668/26345
LA en
DS RISalud
RD Apr 5, 2025