Leigh Syndrome
0
Pipeline Programs
2
Companies
2
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
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
PTC TherapeuticsEPI-743
Taysha Gene TherapiesNone
Clinical Trials (2)
Total enrollment: 30 patients across 2 trials
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Start: Jan 2014Est. completion: Oct 202330 patients
Phase 2Completed
A Study of the Natural Course of SURF1 Deficiency
Start: May 2022Est. completion: May 20220
N/AWithdrawn
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
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.