Late-onset Pompe Disease
Rare Diseases
2
Pipeline Programs
3
Companies
4
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
1
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
BioMarin PharmaceuticalNOVATO, CA
2 programs1
BMN 701Phase 31 trial
Respiratory muscle strength measurements by different techniques.N/A1 trial
Active Trials
Amicus TherapeuticsPA - Philadelphia
1 programSTRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERTN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
BioMarin PharmaceuticalBMN 701
Denali TherapeuticsDNL952
Amicus TherapeuticsSTRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
BioMarin PharmaceuticalRespiratory muscle strength measurements by different techniques.
Clinical Trials (4)
Total enrollment: 32 patients across 4 trials
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
Start: Apr 2014Est. completion: Sep 201624 patients
Phase 3Terminated
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Start: Mar 2026Est. completion: Aug 2028
Phase 1Not Yet Recruiting
NCT03347253Amicus TherapeuticsSTRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Start: Dec 2017Est. completion: Nov 2018
N/ATerminated
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
Start: Oct 2014Est. completion: Jun 20168 patients
N/ATerminated
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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 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space