The Angelman Syndrome Online Registry - A multilingual approach to support global research.
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.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Síndrome de Angelman
Tipo de estudo:
Prognostic_studies
Limite:
Adolescent
/
Adult
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Aged
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Aged80
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Child
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Child, preschool
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Female
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Humans
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Infant
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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