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Our Medicines

Treating the untreatable

Our passion for patients in need fuels our dedication to fill the therapeutic void with hope for the future. With RNA as the basis of our discovery platform, we are able to bring therapeutic options to patients who otherwise would have none.

Our antisense therapies are designed to interact precisely with RNA, allowing us to create treatments that disrupt the disease process and change its course. With three approved best- and first-in-class medicines and 10+ in mid- or late-stage development, our therapeutic breakthroughs are poised to continue to transform the futures of patients for years to come.

For more information about SPINRAZA and U.S. prescribing information, visit

For more information about TEGSEDI and U.S. prescribing information, visit

For WAYLIVRA prescribing information in the EU, please visit

A perpetual and efficient innovation machine

Our platform technology has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases.

Once in a lifetime breakthroughs again and again

Meet Lou and Terry Jo. Dissatisfied with the lack of treatment options for her son with Angelman syndrome, Terry Jo was determined to make the impossible a reality, and found others who shared the same vision.