X-linked Myotubular Myopathy
2
Pipeline Programs
1
Companies
3
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
1
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
1100%
+ 2 programs with unclassified modality
Competitive Landscape
1 companies ranked by most advanced pipeline stage
AstellasChina - Shenyang
3 programs1
1
Resamirigene bilparvovecPhase 2/3Gene Therapy1 trial
ASP2957Phase 1/21 trial
A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) SubjectsN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
AstellasResamirigene bilparvovec
AstellasASP2957
AstellasA Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
Clinical Trials (3)
Total enrollment: 70 patients across 3 trials
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
Start: Aug 2017Est. completion: Mar 203027 patients
Phase 2/3Active Not Recruiting
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
Start: Dec 2025Est. completion: Oct 20279 patients
Phase 1/2Recruiting
A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
Start: Jul 2016Est. completion: Sep 201934 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 70 patients
1 companies competing in this space
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