Patients / Physicians
BioMarin is committed to serving the needs of patients, families and physicians by providing rapid access to therapeutic treatment, disease education and support services.
Following is a listing of diseases related to BioMarin's products. Also listed below are investigational programs now in progress at BioMarin. Click on any of these items to learn more about each disorder. For additional information, visit the Resource Library, or contact BioMarin Patient and Physician Support (BPPS) should you like to receive additional information.
Diseases Treated With BioMarin Products
- Mucopolysaccharidosis I (MPS I)
- Mucopolysaccharidosis IVA (Morquio A syndrome or MPS IVA)
- Mucopolysaccharidosis VI (MPS VI)
- Phenylketonuria (PKU)
- Lambert-Eaton Myasthenic Syndrome (LEMS)*
Programs now in development
- PEG-PAL for PKU
- GILT GAA for Pompe Disease
- PARP Inhibitor for Genetically Defined Cancers
- BMN 111 for Achondroplasia
- BMN 190 for for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease
- BMN 270 AAV-Factor VIII Vector for Hemophilia A
- BMN 250 GILT rhNAGLU for Sanfilippo Syndrome
*Currently approved in the EU only.