Receiving a devastating diagnosis can be overwhelming, especially when there are no approved therapies available and limited information to be found. It is important to know that you are not alone. Through our collaborations with patient advocacy organizations and communities worldwide we work to identify and understand the most urgent needs of people living with severe diseases and their caregivers. Through scientific research and educational resources, we are committed to furthering our understanding of serious and rare diseases, and to offer hope to those communities in need. As experts in living with their disease, the patient and caregiver perspectives inform our approach to research, drug development, and inspire us as we work to get medicines into the hands of those who need it.

Our work with patients and families inspires us, and our commitment to serving them is what fuels us to truly make a positive difference in their lives.

With patient needs as our central focus, we have become the leader in the discovery and development of antisense medicines; RNA-based therapies that allow us to target disease at the source, from the rarest of diseases, to those that impact millions of people.