Rare Disease


Our commitment

澳门葡京赌博游戏的使命是改变罕见病患者的生活. 通过了解患者的独特需求, 澳门葡京赌博游戏可以研究和开发创新药物, 支持获取和倡导罕见病社区.

澳门葡京赌博游戏认为倾听是澳门葡京赌博游戏的责任, understand, and change the lives of patients and those who work tirelessly to help them. Our innovation begins with understanding people living with rare diseases, 是什么推动了澳门葡京赌博游戏所有的努力.


400 million

people around the world are affected by a rare disease, half of whom are children.


rare diseases are known to exist today but more than 90% do not have an approved treatment option.




Alexion’s pioneering legacy in rare diseases is rooted in being the first to translate the complex biology of the complement system into transformative medicines. 通过推动创新研究和开发(R&D)跨越新的疾病目标和模式, we have diversified our pipeline into additional rare diseases over the last several years. Today, 作为澳门葡京网赌游戏的一部分, we are building bridges across our scientific platforms with a focus on bringing more innovative medicines to people worldwide.


Our R&D approach

For 30 years, Alexion’s pioneering science has led to the development of transformational medicines for rare diseases, 今天,澳门葡京赌博游戏继续引领这一潮流. Our success is tied to listening, to understanding, and responding to the communities we serve.

Rare disease R&D presents unique challenges that require continuous innovation to deliver meaningful medicines for patients. Driven by our patient-centric approach, we pave the way by tailoring elements of the R&D process and developing new tools and methods to gain a deeper understanding of many rare conditions and the needs of the people living with them.

Our people

We invest in and value people who believe in the importance of our purpose and understand what it takes to deliver on it. 澳门葡京赌博游戏所做的一切, we are empowered and committed to speak up and perform at our personal best to accelerate our collective impact for people living with rare diseases. 澳门葡京赌博游戏的文化植根于诚信, inclusiveness, and our dedication to joining and supporting the communities in which we live and work.

我很高兴能与Alexion的同事一起发现, develop and deliver medicines that change the lives of people suffering from rare diseases. We also look forward to applying Alexion’s complement biology platform across areas of AstraZeneca’s broader early-stage pipeline, and to making our rare disease medicines available to patients in many countries where AstraZeneca has a strong presence.

Marc Dunoyer, Alexion首席执行官

Join our team

澳门葡京赌博游戏是创新者、领导者和合作者. And at the heart of everything we do is an intense desire to pursue the toughest challenges and truly change the world for people living with rare and devastating diseases.

Our medicines

We cannot provide detailed information about our prescription medicines on this website, 遵守规定.

Our medicines are approved in individual countries for specific uses and the information we provide for patients is governed by local regulations. In some cases, health care professionals and patients can visit local AstraZeneca websites to find out more about our medicines. Please note that in some countries we are not allowed to provide very much, 有时是任何, information on our prescription medicines so you should seek alternative trustworthy sources. 一定要向医疗保健专业人员咨询有关药物的建议.






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We’re building on our fundamental understanding of complement biology and advancing on our core therapy areas of haematology, nephrology, neurology, metabolics, and cardiology. We continue to evolve into new areas where there is a great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.


Phase 1

Phase 1

Phase 2

Phase 2

Phase 3

Phase 3

LCM Projects

LCM Projects