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Clinical trial

BMN 110 US Expanded Access Program

BioMarin PharmaceuticalAPPROVED_FOR_MARKETINGUnited States, Puerto RicoNCT01858103

Abstract

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.

Topics & conditions

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LSD

Keywords

Mucopolysaccharidosis IVA Type AMPS IVA Type AMucopolysaccharidosis IVAMPS IVAMorquio A SyndromeLysosomal Storage DisorderN-acetylgalactosamine-6-sulfataseN-acetylgalactosamine-6-sulfateMucopolysaccharidosis IVAMorquio A SyndromeMPS IVA

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