Lafora Disease
1
Pipeline Programs
2
Companies
2
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Ionis PharmaceuticalsCARLSBAD, CA
1 programNatural History and Functional Status Study of Patients With Lafora DiseaseN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
Elpida TherapeuticsION283
Ionis PharmaceuticalsNatural History and Functional Status Study of Patients With Lafora Disease
Clinical Trials (2)
Total enrollment: 43 patients across 2 trials
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
Start: Dec 2024Est. completion: Oct 202810 patients
Phase 1/2Recruiting
NCT03876522Ionis PharmaceuticalsNatural History and Functional Status Study of Patients With Lafora Disease
Natural History and Functional Status Study of Patients With Lafora Disease
Start: Jan 2019Est. completion: Apr 202233 patients
N/ACompleted
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 actively recruiting trials targeting 43 patients
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.