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More than two decades of people, families and perseverance

In 1997, the company was founded to make a big difference in small patient populations, focusing on giving much-needed attention to the underserved communities of those with rare diseases. 澳门正规博彩十大排行平台 sought to help patients whose voices could not always be heard and treat conditions that were not always in mainstream medical conversations. Throughout its history, 澳门正规博彩十大排行平台 has worked tirelessly to make a difference by pursuing bold science. Our 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.

Since then, we went on to develop and introduce eight first or best-in-class therapies for genetic disorders with limited-to-no treatment options.

Learn more about our history and how we got our start with the short film “Finding Tomorrows.”

Image: A Rare Breed book cover

You can also read more about our legacy with A Rare Breed.

A Rare Breed recounts the first 20 years of 澳门正规博彩十大排行平台’s history, the struggles we faced in developing drugs for little known conditions, and how we grew into a global enterprise. It follows the researchers, patients, and executives who played a role in advancing novel ideas and how they overcame scientific, regulatory, and financial challenges to bring first-in-class and best-in-class therapies to children who needed them.

A Rare Breed is now available on Amazon with all proceeds going to patient advocacy groups focused on rare disease.


Image: 澳门正规博彩十大排行平台 logo
Company Milestones

Look back at the news, announcements and milestones that brought us to where we are today.

Learn More about our company milestones
Our Pipeline

We maximize the impact of genetic innovation to develop transformative therapies.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

Learn More Our Pipeline