Survey of quality of life, phenotypic expression, and response to treatment in Krabbe leukodystrophy

OBJECTIVES: To develop a quality of life (QOL) survey for Krabbe disease (KD), and to thereby improve understanding of its phenotypic expression and response to treatment. METHODS: The survey, the Leukodystrophy Quality of Life Assessment (LQLA) and the Vineland Adaptive Behavior Scales were co‐admi...

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Detalhes bibliográficos
Publicado no:JIMD Rep
Main Authors: Langan, Thomas J., Barczykowski, Amy, Jalal, Kabir, Sherwood, Laura, Allewelt, Heather, Kurtzberg, Joanne, Carter, Randy L.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2019
Assuntos:
Research Reports
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6498827/
https://ncbi.nlm.nih.gov/pubmed/31240167
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12033
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