We have long been known for our expertise in rare diseases, including the class of genetic disorders known as lysosomal storage disorders (LSDs). We have also built a franchise in multiple sclerosis that aims to address the unmet needs of this patient community.
We recognize little information is available about some of these diseases, so we have collected basic information about disease biology, symptoms, and the methods of diagnosis for each disease we treat. This information should be discussed openly and honestly with a health care provider who can help patients and their caregivers understand more about the disease and guide them through the diagnosis and treatment process.
Focusing on uncommon and under-served medical conditions, our Rare Disease therapy group develops breakthrough therapies for patients who might otherwise have few treatment options.
We are committed to becoming a long-term partner to the Multiple Sclerosis community by working to deliver scientific advancements that will have a significant impact on the needs of people living with Multiple Sclerosis.