Juvenile Neuronal Ceroid Lipofuscinosis
1
Pipeline Programs
2
Companies
1
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Clinical Trials (1)
Total enrollment: 39 patients across 1 trials
Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
Start: Mar 2026Est. completion: Mar 202639 patients
Phase 3Not Yet Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs, potential near-term approvals
2 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.