Details of Disease

General Information of Disease (ID: DISBT8KW)

Disease Name Globoid cell leukodystrophy
Disease Class 8A44: Leukodystrophy
Disease Hierarchy
DIS01GPL: Grass pollen hypersensitivity
DISBT8KW: Globoid cell leukodystrophy
ICD Code
ICD-11
ICD-11: 8A44.4

Drug-Interaction Atlas (DIA) of This Disease

Drug-Interaction Atlas (DIA)
This Disease is Treated as An Indication in 1 Clinical Trial Drug(s)
Drug Name Drug ID Highest Status Drug Type REF
PBKR03 DM57HM7 Phase 1/2 Gene therapy [1]
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Molecular Interaction Atlas (MIA) of This Disease

Molecular Interaction Atlas (MIA)

References

1 ClinicalTrials.gov (NCT04771416) A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy). U.S.National Institutes of Health.