Patients & Families

We believe that it is essential to seek a deeper understanding of – and get to know people living with – the serious diseases where we have the potential to make a difference.

Our clinical work starts with science and our belief that our proprietary research platform has the potential restore proteins to slow or stop serious diseases. Our values compel us to go beyond the science, engaging with patients, families and advocates to help us understand the clinical aspects of serious diseases and also the larger impacts of these diseases on patients and their families.

Clinical Studies

Our clinical research in Dravet Syndrome and ADOA is ongoing. Visit our Clinical Studies page to learn more about current and planned studies.

Parisa Sanandaji

Connections that Matter

Patient Advocacy with Passion

“We are dedicated to staying connected with disease communities as we progress our research. This includes hosting patient advocacy organizations to foster open communication, connect with our team, strengthen our partnerships and keep them informed about our progress. We also regularly seek feedback from advocates to inform our work and important decisions that impact patients.”

– Parisa Sanandaji, Head of Global Patient Advocacy

Words and Images Inspire Us to Keep Fighting

Throughout our offices, we display faces and words from fearless patients and their dedicated family members who inspire our work. These remind us every day of just how important it is for us to keep working with urgency to make progress.

Educational Resources

Our support for patients and their families goes beyond our science. We work to provide useful information to the communities we work with. Visit our Educational Resources page to learn more.