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Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice.
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Psychosine, a marker of Krabbe phenotype and treatment effect.
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KrabbeConnect
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KrabbeConnect
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A day in which each patient receives an early diagnosis, has access to state-of-the-art care, and lives a life free of Krabbe disease.