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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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主要な著者: Christina Q. Nguyen, Kristine Alba-Concepcion, Elizabeth E. Palmer, Jackie L. Scully, Nicole Millis, Michelle A. Farrar
フォーマット: Artigo
言語:Inglês
出版事項: BMC 2022-04-01
シリーズ:Orphanet Journal of Rare Diseases
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オンライン・アクセス:https://doi.org/10.1186/s13023-022-02317-6
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