Pompe Disease Infantile-Onset
Rare Diseases
1
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
1
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
SanofiMyozyme
Clinical Trials (2)
Total enrollment: 7 patients across 2 trials
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
Start: Apr 2003Est. completion: Jul 20067 patients
Phase 2Completed
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
N/AAvailable
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On Market— Approved & available
Key Insights
2 companies competing in this space