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The Angelman Syndrome Online Registry - A multilingual approach to support global research.
Krey, Ilona; Heine, Constanze; Frömming, Marcel; Herrmann, Julia; Møller, Rikke S; Weckhuysen, Sarah; Courage, Carolina; Beblo, Skadi; Syrbe, Steffen; Lemke, Johannes R.
Afiliação
  • Krey I; Institute of Human Genetics, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Ilona.Krey@medizin.uni-leipzig.de.
  • Heine C; Institute of Human Genetics, University of Leipzig Medical Center, Leipzig, Germany; Center for Rare Diseases, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Constanze.Heine@medizin.uni-leipzig.de.
  • Frömming M; Institute of Human Genetics, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Marcel.Froemming@medizin.uni-leipzig.de.
  • Herrmann J; Institute of Human Genetics, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Julia.Herrmann@medizin.uni-leipzig.de.
  • Møller RS; Department of Epilepsy Genetics and Personalized Treatment, The Danish Epilepsy Centre, Dianalund, Denmark; Department of Regional Health Research, University of Southern Denmark, Odense, Denmark. Electronic address: rimo@filadelfia.dk.
  • Weckhuysen S; Applied&Translational Neurogenomics Group, VIB Center for Molecular Neurology, VIB, Antwerp, Belgium; Translational Neurosciences, Faculty of Medicine and Health Science, University of Antwerp, Antwerp, Belgium; Department of Neurology, Antwerp University Hospital, Antwerp, Belgium. Electronic a
  • Courage C; Folkhälsan Research Center, Helsinki, Finland. Electronic address: Carolina.Courage@helsinki.fi.
  • Beblo S; Center for Rare Diseases, University of Leipzig Medical Center, Leipzig, Germany; Hospital for Children and Adolescents, Department of Women and Child Health, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Skadi.Beblo@medizin.uni-leipzig.de.
  • Syrbe S; Division of Paediatric Epileptology, Centre for Paediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany. Electronic address: Steffen.Syrbe@med.uni-heidelberg.de.
  • Lemke JR; Institute of Human Genetics, University of Leipzig Medical Center, Leipzig, Germany; Center for Rare Diseases, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: Johannes.Lemke@medizin.uni-leipzig.de.
Eur J Med Genet ; 64(12): 104349, 2021 Dec.
Article em En | MEDLINE | ID: mdl-34619369
In collaboration with the German Angelman syndrome (AS) community, we developed a web-based AS Online Registry to congregate existing as well as future information and scientifically quantify observations made by parents, families and medical professionals. With its user-friendly design as well as its concise and multilingual questionnaire, the registry aims at families who had so far refrained from being recruited by other, more comprehensive and/or English-only, registries. Data can be entered by both parents/families and medical professionals. The study design allows for re-contacting individuals (e.g. to request additional information) enabling collection of longitudinal data. Since its launch in June 2020, more than 200 individuals with AS age 2 month to 83 years have registered and entered their clinical and genetic data. In addition to the German, Turkish, English, Dutch, Italian, Danish and Finnish versions of the registry, we aim for translation into further languages to enable international and user-friendly recruitment of AS individuals. This novel registry will allow for extensive genotype-phenotype correlations and facilitate sharing of de-identified information among clinicians, researchers as well as the Global AS Registry. Furthermore, the registry will allow for identification of individuals suitable for future clinical or pharmacologic trials according to particular genotypic and/or phenotypic properties.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Angelman Tipo de estudo: Prognostic_studies Limite: Adolescent / Adult / Aged / Aged80 / Child / Child, preschool / Female / Humans / Infant / Male País/Região como assunto: Europa Idioma: En Revista: Eur J Med Genet Assunto da revista: GENETICA MEDICA Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Angelman Tipo de estudo: Prognostic_studies Limite: Adolescent / Adult / Aged / Aged80 / Child / Child, preschool / Female / Humans / Infant / Male País/Região como assunto: Europa Idioma: En Revista: Eur J Med Genet Assunto da revista: GENETICA MEDICA Ano de publicação: 2021 Tipo de documento: Article