Precision medicine for genetic childhood movement disorders.

Dev Med Child Neurol

Developmental Neurosciences, UCL Great Ormond Street Hospital, Zayed Centre for Research into Rare Disease in Children, London, UK.

Published: August 2021

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.

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Source
http://dx.doi.org/10.1111/dmcn.14869DOI Listing

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