BioMarin Pharmaceutical has initiated a phase II study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA).

The primary objective of the phase II, open-label, multinational clinical study is to evaluate the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in ten to fifteen MPS IVA patients under five years of age. The secondary objectives are to evaluate urinary keratan sulfate (KS) levels and growth velocity.