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BioMarin Supports Observance of Rare Disease Day February 28

Worldwide Coalition Seeks to Increase Public Awareness and Research to Improve Medical Care for Patients Lacking Adequate Treatment Options

February 28 To Be Observed Around the World With a Variety of Programs and Activities

NOVATO, Calif., Feb. 26 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it will participate in a coalition of organizations led by the National Organization for Rare Disorders (NORD) to support the observance of Rare Disease Day on February 28. A variety of patient organizations, professional societies, government agencies, medical researchers and biopharmaceutical companies will also participate in the campaign, which is designed to raise awareness about the public health issues associated with rare diseases, which affect nearly 30 million Americans and millions of others internationally.

"People with rare diseases remain a medically underserved population around the world," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We support and applaud all of the efforts NORD has made within the last 25 years to spearhead 'orphan' disease legislation and to bring more attention to the unique needs of these patients. Like NORD, we are committed to serving this population, and we will continue to support the organization and its efforts to raise public awareness, increase scientific research and provide these patients with the medical treatments they deserve."

"People with rare diseases face unique challenges compared to those with more common diseases," said NORD President, Peter Saltonstall. "They often have great difficulty obtaining accurate diagnoses and finding medical experts who recognize and understand the complexities of their rare disorders. It is our ongoing mission to work with our partners in industry, science and government to mobilize interest and improve medical care for these patients."

Rare Disease Day activities in the U.S. will include a nationwide network of online activities for patients, caregivers and the general public. The day's events will highlight the unique partnership that exists among industry, medical, research and patient communities and government entities such as the National Institutes of Health (NIH) Office of Rare Diseases and the Food and Drug Administration's (FDA) Office of Orphan Products Development.

February 28 will mark the second annual Rare Disease Day, a concept that was launched in Europe in 2008 by the European Rare Disease Organization (EURODIS), a sister organization to NORD. Rare Disease Day will be observed in the United States, Canada, Europe, Australia and other Pacific regions, and will continue in the hopes of establishing a formalized global Rare Disease Day on the last day of February each year.

A rare disease (also known as an "orphan" disease) is one that affects fewer than 200,000 Americans. According to the NIH there are nearly 7,000 such diseases affecting an estimated 30 million people in the United States.

In 1983, the Orphan Drug Act was passed by Congress to create financial incentives for companies to develop treatments for rare diseases. Since then, approximately 330 orphan drugs and biologics have been approved by the FDA. While estimates indicate that up to 13 million Americans now benefit from these products, more than 15 million patients still remain untreated and underserved.

For more information about Rare Disease Day 2009 activities in the U.S., visit For information about activities worldwide, go to

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of sickle cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 1 clinical development for the treatment of PKU. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

BioMarin(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme(R) is a registered trademark of BioMarin/Genzyme.

About NORD

NORD (, a federation of individuals and patient organizations, was established in 1983 by patient leaders who worked to get the Orphan Drug Act passed. NORD provides advocacy in Washington, D.C. on behalf of the rare disease community and sponsors research grants and fellowships, educational services for patients and medical professionals and other patient assistance programs and public awareness initiatives.


    Investors                                    Media
    Eugenia Shen                                 Susan Berg
    BioMarin Pharmaceutical Inc.                 BioMarin Pharmaceutical Inc.
    (415) 506-6570                               (415) 506-6594

    NORD Media Contact:

    Mary Dunkle
    (203) 744-0100 / (203) 942-6443

SOURCE BioMarin Pharmaceutical Inc.
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