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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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A Journey of Hope: Establishing a Charity for Myotubular Myopathy

This chapter explores the personal journeys of families affected by myotubular myopathy, highlighting the establishment of a charity focused on research and support. It emphasizes the power of collaboration among families, researchers, and stakeholders to drive meaningful change in the treatment of rare genetic disorders. Through heartfelt narratives and discussions about fundraising and clinical trials, the chapter showcases the transformative impact of grassroots efforts in advancing medical research.

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