Our Focus
We look at the whole person.
Connective tissue diseases can affect many parts of the body at once. A patient’s priorities may include the aorta, their vision, and whether they can get through a school day comfortably–all at once. Our proposed therapeutic focus reflects that reality.
We are beginning with Marfan syndrome and related genetic conditions, including Loeys–Dietz syndrome and vascular Ehlers–Danlos syndrome. Different conditions will need different approaches. Patient priorities and scientific evidence will guide our decisions.
Vascular health: more ways to protect life.
Surgery and monitoring have changed the future for many families. We want to help build on that progress with medical treatments that better protect the aorta and other blood vessels. Our goal is to advance therapies that could slow or prevent disease progression, reduce serious complications, and lessen the need for surgery.
Bones, joints, and movement: less pain every day.
Walking between classes. Hanging out with friends. Being productive at work. Getting through the day with energy left to enjoy it. We want to advance treatments for the musculoskeletal problems that can make these ordinary experiences painful or exhausting. Comfort, mobility, and independence belong among the outcomes that matter.
Eyes and vision: preserving how people see their world.
We want better medical treatments for the eye problems associated with these diseases. Preserving sight and reducing the burden of ocular complications are part of our vision for a better life.
These priorities will shape our search for therapeutic opportunities. We will assess both existing medicines that might have a new use and new approaches that could change the course of disease.