European Galactosemia Network Patient Registry (GalNET) (Version 1)
Research type
Research Study
Full title
European registry and network for Galactosemias (GalNET)
IRAS ID
188818
Contact name
Stephanie Grunewald
Contact email
Sponsor organisation
Maastricht University Medical Centre
Duration of Study in the UK
3 years, 1 months, 30 days
Research summary
Galactosemias are rare, challenging disorders of galactose metabolism that may have a fatal outcome. The current treatment of these diseases: a galactose restricted diet, although lifesaving in the newborn, fails to prevent cognitive, motor and fertility impairments. Because these diseases are rare, single centres base patient care on very limited experience, which leads to sub-optimal care and the adoption of varying approaches towards treatment and follow-up of patients between different countries. In 2012, the European Galactosemia Network (EGN) was established. It focuses on the advancement of research, diagnosis and treatment of these conditions by close collaboration between clinicians, scientists and patient associations. Our mission is to improve the quality of care and access to multi-disciplinary expertise for European patients affected by the galactosemia group of disorders. One of the objectives of the EGN is the development and maintenance of a European Galactosemia patient registry for the collection of historical clinical data and recruitment of patients into clinical trials. A European registry for patients will allow us to delineate the medical history of dietary treated patients and answer scientific questions to develop care pathways.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
17/YH/0331
Date of REC Opinion
13 Dec 2017
REC opinion
Further Information Favourable Opinion