Neuromuscular Disease
2
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
2
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2024
2025
2026
2027
2028
Intellia Therapeuticsnexiguran ziclumeran
Clinical Trials (1)
Total enrollment: 50 patients across 1 trials
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Start: Nov 2024Est. completion: Aug 202850 patients
Phase 3Active Not 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
2 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space