International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study
Abstract 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...
Guardado en:
Autores principales: | William Evans, Marc Patterson, Frances Platt, Christina Guldberg, Toni Mathieson, Jessica Pacey, the Core Working Group for the Delphi Study |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
BMC
2021
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Materias: | |
Acceso en línea: | https://doaj.org/article/46cfd0e5c0d2451ea0832be576c10057 |
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