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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
7
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Children's Hospital of Oakland
Oakland, California, United States
Children's Hospital of Orange Country
Orange, California, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey, United States
The objective of this extension study was to monitor the long-term safety and efficacy
Time frame: 3 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
New York University [NYU] School of Medicine
New York, New York, United States
Pediatrique Hospital Debrousse
Lyon, France
Hôpital Porte Madeleine
Orléans, France
The Morningside Clinic
Johannesburg, South Africa