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Precision medicine for genetic childhood movement disorders.
Soo, Audrey K S; Ferrini, Arianna; Kurian, Manju A.
Afiliação
  • Soo AKS; Developmental Neurosciences, UCL Great Ormond Street Hospital, Zayed Centre for Research into Rare Disease in Children, London, UK.
  • Ferrini A; Department of Paediatric Neurology, Great Ormond Street Hospital, London, UK.
  • Kurian MA; Developmental Neurosciences, UCL Great Ormond Street Hospital, Zayed Centre for Research into Rare Disease in Children, London, UK.
Dev Med Child Neurol ; 63(8): 925-933, 2021 08.
Article em En | MEDLINE | ID: mdl-33763868
Increasingly effective targeted precision medicine is either already available or in development for a number of genetic childhood movement disorders. Patient-centred, personalized approaches include the repurposing of existing treatments for specific conditions and the development of novel therapies that target the underlying genetic defect or disease mechanism. In tandem with these scientific advances, close collaboration between clinicians, researchers, affected families, and stakeholders in the wider community will be key to successfully delivering such precision therapies to children with movement disorders. What this paper adds Precision medicine for genetic childhood movement disorders is developing rapidly. Accurate diagnosis, disease-specific outcome measures, and collaborative multidisciplinary work will accelerate the progress of such strategies.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Medicina de Precisão / Transtornos dos Movimentos Tipo de estudo: Prognostic_studies / Screening_studies Limite: Child / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Medicina de Precisão / Transtornos dos Movimentos Tipo de estudo: Prognostic_studies / Screening_studies Limite: Child / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article