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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Detalles Bibliográficos
Autores principales: Langan, Thomas J., Barczykowski, Amy, Jalal, Kabir, Sherwood, Laura, Allewelt, Heather, Kurtzberg, Joanne, Carter, Randy L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2019
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6498827/
https://www.ncbi.nlm.nih.gov/pubmed/31240167
http://dx.doi.org/10.1002/jmd2.12033

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