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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.

病人 Stories

Meet some of the exceptional people whose stories remain at the heart of everything 澳门正规博彩十大排行平台所做的.

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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.

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Image: 病人, Juanito
病人 & Community 支持

Treatment is only the beginning.

We are committed to the communities we treat beyond the therapies we develop.

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