About Us

Transforming Lives Through Genetic Discovery

Over two decades ago when we first opened our doors, we focused on giving much-needed attention to the underserved communities of those with rare diseases. These rare disease communities mostly affected children and were often ignored. At the time, 澳门正规博彩十大排行平台 developed the only treatments for these life-altering conditions, giving hope to patients and families.

Throughout our history, we’ve worked tirelessly to make a difference by pursuing bold science while respecting, educating, and connecting with patients. Through our expertise in genetics and molecular biology, we have been able to develop targeted therapies that address the root cause of the exact conditions we seek to treat.

Our discoveries have led us to countless breakthroughs, best-in-class treatments and many ‘firsts’ in the category. We are grateful to able to better the lives of those struggling with genetic diseases.

As we look forward, we strive to make an even greater impact by applying the same science-driven, patient-forward approach to a broader range of genetic disorders.

The more innovative solutions we develop, the more lives we can impact.

This isn’t just our passion, it’s our purpose.