Unlocking Tomorrows Together
In the last 25 years, 澳门正规博彩十大排行平台 has worked hard to make a difference, paving unchartered territories, and bettering the lives of those suffering from rare genetic disease. The company’s first approved therapy was inspired by a father who refused to accept that his son’s rare diagnosis lacked a viable therapy. That relentless pursuit to change the course of his son’s future through the power of treatment is at the core of who 澳门正规博彩十大排行平台 is today.
Learn more about how our patients inspire, drive and shape our work at 澳门正规博彩十大排行平台.
Meet some of the exceptional people whose stories remain at the heart of everything we do.Learn More about our patients ⌃
Diseases & Conditions
Through our unparalleled expertise in genetics and molecular biology, we develop targeted therapies that address the root cause of the conditions we seek to treat.
For each disease state we seek to treat, there are a number of organizations and resources that provide great insight and support services.Learn More about our resources ⌃