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Dr Ana Lisa Tavares, Anne Lennox, Dr Meriel McEntagart, Dr Carlo Rinaldi: Can patient collaboration shape the future of therapies for rare conditions?

Behind the Genes

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Empowering Patient Voices in Rare Condition Research

This chapter highlights the critical role of patient collaboration and insights from families in addressing rare conditions. It advocates for enhanced research funding and explores how early gene therapy trials can inform future treatment innovations.

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