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, r...
| Autores principales: | , , , , , |
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| Otros Autores: | |
| Formato: | Journal article |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2021
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