Patients and 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.
The following is a list of diseases related to BioMarin’s products. Also listed below are investigational programs now in progress at BioMarin. Click on any of these links to learn more about each disorder. For additional information, please visit our Resource Library, or contact BioMarin Patient and Physician Support 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
- GALNS for MPS IVA
- IGF2-GAA for Pompe Disease
- Drisapersen for Duchenne Muscular Dystrophy
- BMN 044 for Duchenne Muscular Dystrophy
- BMN 045 for Duchenne Muscular Dystrophy
- BMN 053 for Duchenne Muscular Dystrophy
*Currently approved in the EU only.