RTW

There are over 10,000 rare diseases, yet less than 5% of them have effective treatments. We collaborate with research teams, families, and advocacy organizations from around the world to work toward treatments and improve patient access to affordable care.

We are committed to supporting underserved communities. We build partnerships with community-based organizations to respond to the urgent public health needs of our neighbors. 

In addition to providing funding to labs conducting research on rare diseases, we have our own lab dedicated to advancing promising therapeutics. This work is driven by the promise of patient impact, not financial returns.

Science has incredible potential to impact lives, but without the right policies and education these innovations may never reach patients. Through education and advocacy, we aim to support equitable access to medical innovation for patients around the world.