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The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review

Abstract Background The patient voice is becoming increasingly prominent across all stages of therapeutic innovation. It pervades research domains from funding and recruitment, to translation, care, and support. Advances in genomic technologies have facilitated novel breakthrough therapies, whose gl...

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Main Authors: Christina Q. Nguyen, Kristine Alba-Concepcion, Elizabeth E. Palmer, Jackie L. Scully, Nicole Millis, Michelle A. Farrar
Formáid: Artigo
Teanga:Inglês
Foilsithe: BMC 2022-04-01
Sraith:Orphanet Journal of Rare Diseases
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Rochtain Ar Líne:https://doi.org/10.1186/s13023-022-02317-6
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