Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

NCT00976352 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

9

Enrollment

OTHER

Sponsor class

Conditions

Pompe Disease

Interventions

DRUG: rAAV1-CMV-GAA (study agent) Administration
OTHER: RMST

Sponsor

University of Florida

Collaborators

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