Facioscapulohumeral Muscular Dystrophy
3
Pipeline Programs
3
Companies
4
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
2
1
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Merck & Co.ACE-083
aTyr PharmaATYR1940
aTyr PharmaATYR1940
Dyne TherapeuticsFSHD-specific functional rating scale
Clinical Trials (4)
Total enrollment: 17 patients across 4 trials
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
Start: May 2019Est. completion: Mar 2020
Phase 2Terminated
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
Start: Jul 2016Est. completion: Apr 20178 patients
Phase 1/2Completed
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Start: Aug 2015Est. completion: May 20169 patients
Phase 1/2Completed
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
Start: Mar 2018Est. completion: Dec 2027
N/ARecruiting
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 17 patients
3 companies competing in this space