Explore / Clinical trial
Clinical trial

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

BioMarin PharmaceuticalTerminatedPhase 2Started Aug 1, 201216 enrolledUnited States, Germany, United KingdomNCT01697319

Abstract

The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Topics & conditions

Detected automatically by Psychedelic Commons from the title and abstract, not labels from the source. They may be incomplete.

LSD

Keywords

Mucopolysaccharidosis IVA Type AMPS IVA Type AMucopolysaccharidosis IVAMPS IVAMorquio A SyndromeLysosomal Storage DisorderN-acetylgalactosamine-6-sulfataseN-acetylgalactosamine-6-sulfatesulfatasegalactose-6-sulfataseGALNSenzyme replacement therapyERTMOR-006CPET

Psychedelic Commons aggregates metadata from open sources and links back to the original. Rights remain with the authors and publishers. Something wrong with this record? Report a problem.