Giugliani R

Language: Spanish
References: 3
Page: 329-330
PDF size: 45.55 Kb.

ABSTRACT

In the subject of rare diseases, experience and knowledge is limited. With a rare disease registry, longitudinal data can be added to increase information of them and to improve the quality of medical care and the patient outcome. Registries, unlike randomized controlled trials, provide a source of real-time information to develop guidelines for monitoring and to support patient management.
Registries are essential to show the real situation of rare diseases and to establish management goals in order to address the unmet needs of the patients.

REFERENCES

1. Clarke JT, Giugliani R, Sunder-Plassmann G, Elliott PM, Pintos-Morell G, Hernberg-Ståhl E, Malmenäs M, Beck M; FOS Investigators. Impact of measures to enhance the value of observational surveys in rare diseases: the Fabry Outcome Survey (FOS). Value Health 2011;14(6):862-6.

2. Jones S, James E, Prasad S. Disease registries and outcomes research in children: focus on lysosomal storage disorders. Paediatr Drugs 2011;13(1):33-47.

3. Cole JA, Taylor JS, Hangartner TN, Weinreb NJ, Mistry PK, Khan A. Reducing selection bias in case-control studies from rare disease registries. Orphanet J Rare Dis 2011;6:61.