持久的目标，扩大的范围

澳门正规博彩十大排行平台 was founded in 1997 to make a big difference in small patient populations. In the years that followed, we have established a significant foundation for growth. 走自己的路带来了无数的突破, 改善罕见遗传病患者的生活.

当澳门正规博彩十大排行平台回顾过去四分之一世纪的进步时, we take this opportunity to also look forward to what’s next for 澳门正规博彩十大排行平台. 在澳门正规博彩十大排行平台不断追求对患者影响最大化的过程中, 澳门正规博彩十大排行平台不断问自己:澳门正规博彩十大排行平台还能做些什么? The outcome of this ongoing self-assessment has helped bring clarity to how we can best deliver on our purpose – 通过基因发现来改变生命.

同样的方法，更大的影响

澳门正规博彩十大排行平台由科学推动, patient-forward approach has propelled our last 25 years of drug development and will continue to do so for the next 25 years and beyond. Our historic commitment to rare and ultra-rare communities remains unwavering, 同时使澳门正规博彩十大排行平台能够在其他患者群体中做更多的事情. 展望未来, we will continue to apply our approach in rare diseases while also expanding to larger genetic disorders and genetic subsets of more common conditions to make an even greater impact.

Applying our knowledge to make a transformative impact is not just a calling, 而是对那些受益最大的人的义务. 每一次成功, we recognize that “those who will benefit most” is an ever-expanding group of individuals for whom we work every day. 像这样, our expansion beyond rare diseases is a means to further deliver on our deep-rooted and continuing commitment to patients.

由科学推动

在一起, our four core attributes enable our people to achieve their full potential and the full potential of our science.

1. 遗传学: We have unparalleled expertise in genetics and molecular biology and a clear understanding of underlying disease mechanism. 在一起, this enables us to explore diverse modalities and discover the best approach that will yield the greatest effect.
2. 可估价的: To yield the greatest effect we must first be able to adequately evaluate our progress. 在设计研究时, we use readily assessable endpoints that offer a clear efficacy signal and reliability translate into clinical benefit for patients.
3. 有针对性的: We work tirelessly to develop therapies that directly or proximally address the root cause of genetic disorders and genetic subsets of other conditions – whether common or rare.
4. 转型: Regardless of which condition we aim to treat, we will never settle for a marginal effect. Every step we take is to maximize impact on patients’ lives by profoundly improving the way they feel, 函数, 和生存.

病人-Forward

The people of 澳门正规博彩十大排行平台 are proof that science and emotion are not mutually exclusive. We are on a dispassionate search for scientific truth while driven by the emotional connection and commitment we have forged with patients.

The resulting sense of urgency is what propels us forward every day. 它激励澳门正规博彩十大排行平台最大限度地发挥基因创新的影响. 它给了澳门正规博彩十大排行平台好奇的勇气. It encourages us to adapt our ways of working to optimize productivity, efficiency, and focus. 它迫使澳门正规博彩十大排行平台每天追求澳门正规博彩十大排行平台的目标.

The end goal has always been better lives and now we can reach more. 澳门正规博彩十大排行平台接触的人越多，澳门正规博彩十大排行平台的影响力就越大.