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Better Medicines for Children: Lessons Learnt and Share Learnings at the EFGCP Annual Paediatric Conferences

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Using facial reaction analysis and machine learning to objectively assess palatability of medicines in children

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Feasibility of an App-Assisted and Home-Based Video Version of the Timed up and Go Test for Patients with Parkinson’s Disease: vTUG

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Video-based assessments of activities of daily living: generating real-world evidence in pediatric rare diseases

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The Digital 1-Minute Walk Test: A New Patient-centered Cardiorespiratory Endpoint

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Recommendations to address respondent burden associated with PROs

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A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH)

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Development and usability testing of an ePRO solution for patients with inflammatory diseases in an ATMP basket trial

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A pilot study of a digital 1-Minute Walk Test as a novel decentralised endpoint in pulmonary hypertension research

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SARAhome video assessment deployed in pan-European ataxia observational study

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Accelerometer-derived sleep measures in idiopathic dystonia

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A global neuronopathic Gaucher disease registry (GaRDIAN): a patient-led initiative

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Considerations for patient and public involvement and engagement in health research

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Digitally enabled decentralised research: opportunities to improve the efficiency of clinical trials and observational studies

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Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases