Innovative therapeutics

Patients/
Physicians

Diseases

BioMarin provides marketed therapies to patients with PKU, MPS I, MPS VI, and LEMS and the company continues to work at a rapid pace to develop additional drugs for other patients suffering from a variety of rare genetic diseases.

The following is a list of rare genetic diseases being addressed at BioMarin. To learn more about each product development program, click on selected links. You may also visit the Resource Library, or contact BioMarin RareConnections should you like to receive additional information.

Lysosomal Storage Disorders
MPS I
Morquio A (MPS IVA)
MPS VI
PKU
LEMS

Read our patient stories…

liamside

ginaside

Kendra - BioMarin Patient Stories

Watch our patient videos…

Kendra - video

Annabelle - video

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS

Products

Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

Contact Global Medical Information

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