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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Bibliographic Details
Main Authors: Christina Q. Nguyen, Kristine Alba-Concepcion, Elizabeth E. Palmer, Jackie L. Scully, Nicole Millis, Michelle A. Farrar
Format: Artigo
Language:Inglês
Published: BMC 2022-04-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Advanced therapeutics
Paediatric neurology
Patient advocacy
Precision medicine
Online Access:https://doi.org/10.1186/s13023-022-02317-6
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