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Enzyme Replacement Therapy

1 for Pompe Disease

Phase 2
Waitlist Available
Research Sponsored by Genzyme, a Sanofi Company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Summary

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

Eligible Conditions
  • Pompe Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alglucosidase alfa
FDA approved

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
526 Previous Clinical Trials
185,613 Total Patients Enrolled
Medical MonitorStudy DirectorGenzyme, a Sanofi Company
1,673 Previous Clinical Trials
988,428 Total Patients Enrolled
~0 spots leftby Sep 2025
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