Clinical Trial: BMN 110 US Expanded Access Program

Study Status: Approved for marketing
Recruit Status: Approved for marketing
Study Type: Expanded Access



Official Title: A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA

Brief Summary:

The Expanded Access Program (EAP) is an open-label, multicenter program to:

  1. Provide patients who have been diagnosed with Mucopolysaccharidosis IVA (MPS IVA) access to BMN 110 until commercial product is available
  2. Collect additional information on the safety and tolerability of BMN 110 administration in patients with MPS IVA

Patients enrolled in the EAP will receive 2.0 mg/kg intravenous infusions of BMN 110 every week during the program.