Details of Disease | DrugMAP

General Information of Disease (ID: DISAH2NN)

Disease Name	CLN3 Batten disease
Disease Class	5C56: Lysosomal disease
Disease Hierarchy	DIS01GPL: Grass pollen hypersensitivity DISAH2NN: CLN3 Batten disease
ICD Code	ICD-11 ICD-11: 5C56

Drug-Interaction Atlas (DIA) of This Disease

Drug-Interaction Atlas (DIA)

This Disease is Treated as An Indication in 2 Clinical Trial Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
PLX-200	DMFCF7J	Phase 3	Small molecule	[1]
AT-GTX-502	DMR19UD	Phase 1/2	Gene therapy	[2]
------------------------------------------------------------------------------------

Molecular Interaction Atlas (MIA) of This Disease

Molecular Interaction Atlas (MIA)

References

1	ClinicalTrials.gov (NCT04637282) A Randomized. Multicenter, Double-Blind, Placebo-Controlled Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease. U.S.National Institutes of Health.
2	ClinicalTrials.gov (NCT03770572) Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9. U.S.National Institutes of Health.