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Patients & Community

Our pursuit to support patients is limitless

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.

Supporting patients relentlessly

We are committed to helping patients stay connected and informed.

Effecting change through community

In our pursuit to create a brighter future for patients, we must partner and collaborate to change the healthcare landscape, increase disease awareness and drive innovation.

Once in a lifetime breakthroughs again and again

Meet Fred and Lynne. For almost 40 years, the couple did not have a name for Fred’s debilitating condition. Finally, now when they hear familial chylomicronemia syndrome, there’s hope, and a promising path forward.