Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.BioMarin Pharmaceutical has initiated a Phase III study, which will evaluate N-acetylgalactosamine 6-sulfatase (GALNS) as a treatment for the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also known as Morquio A Syndrome.
Subscribe to our email newsletter
The randomized, double-blind, placebo-controlled Phase III trial will evaluate the safety and efficacy of GALNS in patients with MPS IVA.
In the study, patients will receive doses of 2mg/kg/week and 2mg/kg/every other week for a treatment period of 24 weeks.
BioMarin said that the primary endpoint will be the six-minute walk test, and the secondary endpoints will be the three-minute stair climb test and urine keratan sulfate concentration.
The study will be conducted at approximately 40 centres worldwide and will enroll 160 patients.