International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study

BACKGROUND: Several scales have been developed in the past two decades to evaluate Niemann–Pick disease Type C (NPC) severity in clinical practice and trials. However, a lack of clarity concerning which scale to use in each setting is preventing the use of standardised assessments across the world,...

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Detalles Bibliográficos
Autores principales: Evans, William, Patterson, Marc, Platt, Frances, Guldberg, Christina, Mathieson, Toni, Pacey, Jessica
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8600786/
https://www.ncbi.nlm.nih.gov/pubmed/34794481
http://dx.doi.org/10.1186/s13023-021-02115-6

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