Patient Advocacy & Engagement

At BioMarin, we have been focused on treating rare diseases and conditions for more than 20 years and we have been doing it through close collaboration with patients and patient communities. We recognise the strategic value and the importance of integrating the patient voice across the entire process, starting at research and development and continuing systematically even after a medicine is approved to best serve unique needs of patients and their families. Our Patient Advocacy and Engagement teams collaborate with a broad range of patient communities across multiple geographies to make sure that patients’ voice is integrated into everything we do.

Patient Organisations

Learn more about some of the patient advocacy and support groups in the disease areas where we work.

Mucopolysaccharidosis (MPS):

MPS Network
Haemophilia:

EHC – European Haemophilia Consortium
Hereditary Angioedema (HAE):

HAEi – HAE International
Duchenne Muscular Dystrophy (DMD):

World Duchenne Organization
Rare Diseases:

EURORDIS – Rare Diseases Europe

Community Awareness Days

Learn more about some of the awareness days organised by the patient community.

Rare Disease Day – 28th/29th February
World Hemophilia Day – 17th April
International MPS Day – 15th May
hae day :–) – 16th May
International Batten Disease Awareness Day – 9th June
International PKU Day – 28th June
Duchenne Awareness Day – 7th September
International Dwarfism Awareness Day – 25th October

Patient Advocacy & Engagement