Innovative therapeutics

Independent Research

In alignment with BioMarin’s commitment to scientific innovation and areas of unmet medical need, BioMarin may provide product and funding support for independent research in rare diseases and genetic disorders.

This program supports the following types of Investigator Initiated Studies:

  • Clinical studies of approved and investigational uses of BioMarin medicines, including medicines approved by the FDA or still in development (i.e. interventional studies phase I to IV).
  • Clinical observational studies, real world evidence (i.e. non-interventional studies)
  • Clinical studies requesting BioMarin product only

This program does not provide support for animal model studies, CME or non-CME educational activities, event sponsorships, or charitable contributions.

Our medical science staff located throughout the globe are available to guide you through each step in the application process. If you have a concept you would like to submit, please reach out to your Medical Science Liaison (MSL) or Area Medical Director. If you don’t yet have a medical staff contact or for general inquiries, please email us at: independentresearch@bmrn.com

MY INDEPENDENT RESEARCH APPLICATIONS

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QUESTIONS / COMMENTS

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU

Products

PALYNZIQ®
Brineura®
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®

Patient/Physician Support Contact Information

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

Global Medical Information

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