Oral: Special Session: Paediatric Drug Development

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Oral: Special Session: Paediatric Drug Development

Population pharmacokinetic analysis of guanabenz in children with Vanishing White Matter: Evaluating scaling adult drug safety and pharmacokinetics data

Thursday 16 October, 2025

Introduction: Vanishing White Matter (VWM) is an inherited leukodystrophy caused by mutations that affect the eIF2B protein, which has a…

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Extrapolating treatment to children less than 6 years old: the successful story of velmanase alfa in alpha-mannosidosis

Thursday 16 October, 2025

Objectives: Alpha-mannosidosis (AM) is an ultra-rare disorder caused by the deficiency of the lysosomal enzyme alpha-mannosidase. It can manifest at…

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Paediatric Extrapolation – Opportunities and Challenges

Thursday 16 October, 2025

In recent years paediatric extrapolation has been brought forward as a key approach to modernise paediatric drug development. In the…

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