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24 mai 2023
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- handle: 10670/1.c7fjne
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Rare disorders Uncommon diseasesCiter ce document
bartosb, « Gene-targeted therapies for rare diseases: How to achieve equitable access », ELSIBI, ID : 10670/1.c7fjne
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A new commentary published in the American Journal of Medical Genetics investigates the role equitable access has to play in allowing gene-targeted therapies to live up to their potential for addressing rare diseases. Gene-targeted therapies are those that make use of knowledge of specific genetic aetiologies or disease-causing variants to better treat disorders with a genetic basis. As many rare diseases fall into this category, they have the potential to transform care for rare diseases. Ho...