Resource Library
Access publications, videos, websites and other up-to-date information about our products and therapy areas.
BioMarin is committed to supporting healthcare professionals with a range of information and resources to assist in the management of patients with rare diseases.
Achondroplasia is a rare genetic bone growth disorder and the most common form of disproportionate short stature. It is caused by a change in the fibroblast growth factor receptor 3 (FGFR3) gene, which impairs the growth of bone in the cartilage of the growth plate.
Phenylketonuria (PKU) is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH).
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease is an ultra-rare and rapidly progressive paediatric brain disorder and one of the most common forms of neuronal ceroid lipofuscinosis, a group of inherited disorders collectively known as Batten disease.
Mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome, is an ultra-rare inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B), an enzyme required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs).
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an ultra-rare inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine-6-sulfatase (GALNS), an enzyme required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs).
Please select a product to access further information.
BRINEURA® is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
KUVAN® is indicated for the treatment of hyperphenylalaninemia (HPA) in sapropterin-responsive adult and paediatric patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
NAGLAZYME® is indicated for the treatment of Mucopolysaccharidosis type VI (MPS VI) storage disorder, a disease in which the enzyme level of Nacetylgalactosamine 4-sulfatase is absent or lower than normal.
PALYNZIQ® is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 and older, whose blood phenylalanine levels cannot be adequately controlled by other means such as by diet.
VIMIZIM® is indicated for the treatment of MPS IVA (mucopolysaccharidosis type IVA, morquio A syndrome).
VOXZOGO® is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
Access publications, videos, websites and other up-to-date information about our products and therapy areas.
BioMarin Pharmaceutical Australia Pty Ltd
119 Willoughby Rd, Crows Nest, NSW 2065, Australia
Email: anz@bmrn.com
Registered pharmacies and hospitals only:
Patients and consumers should speak to their community pharmacy or prescriber for more information.
Australia
DHL Direct to Market
Toll Free: 1800 077 421 (Option 1)
Email: d2mcs@dhl.com
New Zealand
Healthcare Logistics
Ph: 09 969 0734
E-mail: orders@healthcarelogistics.co.nz
Hours: 24 hrs/day, 7 days/week
Email: Medinfo@bmrn.com
Australia
Toll Free: 1800 387 876
Local: 02 8520 3255
New Zealand
Toll Free: 0800 882 012
Local: 09 801 1051
Hours: 24 hrs/day, 7 days/week
Email: Medinfo@bmrn.com
Submit an Adverse Event ›
Australia
Toll Free: 1800 387 876
Local: 02 8520 3255
New Zealand
Toll free: 0800 882 012
Local: 09 801 1051
TGA adverse event reporting
Website: www.tga.gov.au/reporting-problems
Registered pharmacies and hospitals only:
Australia
DHL Direct to Market
Toll Free: 1800 077 421 (Option 3)
Email: d2mcs@dhl.com
New Zealand
Healthcare Logistics
Ph: 09 969 0734
Email: orders@healthcarelogistics.co.nz
Email: anz@bmrn.com
Email: ir@bmrn.com
Tel: +1 (415) 455-7558