The 6,000 to 8,000 rare diseases are characterized by a broad diversity of disorders and symptoms.
Our Rare Disease business is focused on products for the treatment of rare genetic diseases and other chronic debilitating diseases, including Lysosomal Storage Disorders, or LSDs, a group of metabolic disorders caused by enzyme deficiencies. Our principle rare disease products are enzyme replacement therapies:
1. For more information, please consult the Summaries of Product Characteristics for the products available in your country.
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