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

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

Abstract 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...

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Bibliographic Details
Main Authors: Thomas J. Langan, Amy Barczykowski, Kabir Jalal, Laura Sherwood, Heather Allewelt, Joanne Kurtzberg, Randy L. Carter
Format: Article
Language:English
Published: Wiley 2019-05-01
Series:JIMD Reports
Subjects:
neurodegeneration
newborn screening
transplantation
Online Access:https://doi.org/10.1002/jmd2.12033

Internet

https://doi.org/10.1002/jmd2.12033

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