Infantile Krabbe disease (0–12 months), progression, and recommended endpoints for clinical trials
Abstract Objective Krabbe disease is due to deficiency of galactocerebrosidase, resulting in progressive neurodegeneration due to demyelination. The purpose of this study is to document disease progression in the newly classified infantile‐onset (0–12 months). We evaluated the outcomes of hematopoie...
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| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2024-12-01
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| Series: | Annals of Clinical and Translational Neurology |
| Online Access: | https://doi.org/10.1002/acn3.52114 |
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