Rare Disease

Transforming the lives of people affected by rare diseases

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Our commitment

澳门葡京赌博游戏的使命是通过了解罕见病患者的独特需求来改变他们的生活, we can research and develop innovative medicines, support access and advocate for the rare disease community.
 

We believe it is our responsibility to listen to, understand, 改变病人和那些不知疲倦地帮助他们的人的生活. 澳门葡京赌博游戏的创新始于了解患有罕见疾病的人, which fuels all of our efforts.

Rare diseases by the numbers

400 million

people around the world are living with a rare disease1

10,000

目前已知存在罕见病，但90%以上的罕见病没有获得批准的治疗方案2,3

5+ years

要获得准确的诊断，一条包括多位医生、专家和误诊的道路4

Our strategy in Rare Disease

Alexion在罕见疾病方面的开创性遗产植根于成为第一个将补体系统的复杂生物学转化为变革性药物的公司. By driving innovative research and development (R&D) across new disease targets and modalities, 在过去的几年里，澳门葡京赌博游戏已经将澳门葡京赌博游戏的产品线多样化到其他罕见疾病. Today, as part of AstraZeneca, 澳门葡京赌博游戏正在澳门葡京赌博游戏的科学平台之间搭建桥梁，重点是为全世界的人们带来更多创新药物.

Our rare disease strategy focuses on three core priorities:

1. 通过制定智能和有效的战略来加速患者获得澳门葡京赌博游戏的药物.

2. Innovate by investing in science, platforms and capabilities, including using our own technologies and research capabilities.

3. 超越澳门葡京赌博游戏目前的地理覆盖范围，帮助尽可能多的罕见疾病患者.

Rare disease news and stories

Clinical trial innovation in rare disease
Driving health equity for the rare disease community

Our R&D approach

For 30 years, 亚历克森的开创性科学导致了罕见疾病变革性药物的发展, and we continue to lead the way today. 澳门葡京赌博游戏的成功与倾听、理解和回应澳门葡京赌博游戏所服务的社区息息相关.

Rare disease R&D提出了独特的挑战，需要不断创新才能为患者提供有意义的药物. 在澳门葡京赌博游戏以患者为中心的方法的推动下，澳门葡京赌博游戏通过定制R的元素铺平了道路&D过程和开发新的工具和方法，以更深入地了解许多罕见的疾病和与他们一起生活的人的需求.

Our people

澳门葡京赌博游戏投资并重视那些相信澳门葡京赌博游戏的目标的重要性并理解如何实现它的人. In everything we do, 澳门葡京赌博游戏有能力并承诺尽澳门葡京赌博游戏个人最大努力大声疾呼，加快澳门葡京赌博游戏对罕见病患者的集体影响. Our culture is rooted in integrity, inclusiveness, 澳门葡京赌博游戏致力于加入和支持澳门葡京赌博游戏生活和工作的社区.

I am delighted to work with Alexion colleagues to discover, 开发和提供药物，改变罕见病患者的生活. 澳门葡京赌博游戏也期待将Alexion的补充生物学平台应用于澳门葡京网赌游戏更广泛的早期产品线领域, 让澳门葡京赌博游戏的罕见疾病药物能够在澳门葡京网赌游戏拥有强大影响力的许多国家提供给患者.

Marc Dunoyer, 澳门葡京网赌游戏(AstraZeneca)首席战略官
Lisa Hickey

Vice President, US Business Unit Lead Neurology

View Lisa's profile
Judy Campagnari

Executive Director, Head of Alliance and Integration Team (AIT)

View Judy's profile

Join our team

We are innovators, leaders and collaborators. 澳门葡京赌博游戏所做的一切的核心是一种强烈的愿望，即追求最艰巨的挑战，为患有罕见和毁灭性疾病的人真正改变世界.

Alexion careers website

Our medicines

澳门葡京赌博游戏无法在本网站上提供澳门葡京赌博游戏处方药的详细信息, in compliance with regulations.

澳门葡京赌博游戏的药物在个别国家获得了特定用途的批准，澳门葡京赌博游戏为患者提供的信息受当地法规的约束. In some cases, 医疗保健专业人员和患者可以访问当地的澳门葡京网赌游戏网站，了解更多澳门葡京网赌游戏药物的信息. 请注意，在一些国家，澳门葡京赌博游戏不允许提供太多, or sometimes any, 澳门葡京网赌游戏处方药的信息，所以你应该寻找其他值得信赖的来源. Always ask a healthcare professional for advice about medicines.

Find your country site in the AZ Network
Our full portfolio of medicines

Our pipeline for Rare Disease

Advancing our pipeline every day

澳门葡京赌博游戏正在建立对补体生物学的基本理解，并在血液学的核心治疗领域取得进展, nephrology, neurology, metabolics, and cardiology. 澳门葡京赌博游戏继续向新的领域发展，在这些领域有很大的未满足的需求和机会，帮助患有罕见疾病的人及其家人充分过上最好的生活.

第三阶段/LCM项目:指的是第二/第三阶段的关键资产, or that have been submitted for regulatory approval, 并且可能包括现在在一个或多个主要市场推出的资产(在所有适用的主要市场推出时删除).

Our full Rare Disease pipeline

References

1. Rare disease facts [Internet]. 2024 [cited 2024 Jan 17]. Available from: http://globalgenes.org/rare-disease-facts/

2. Fermaglich LJ, Miller KL. 对孤儿药指定和批准的罕见疾病和病症的综合研究超过了《澳门葡京网赌游戏》的四十年. Orphanet Journal of Rare Diseases. 2023 Jun 23;18(1). doi:10.1186/s13023-023-02790-7

3. About [Internet]. U.S. Department of Health and Human Services; [cited 2024 Jan 17]. Available from: http://rarediseases.info.nih.gov/about

4. Barriers to Rare Disease Diagnosis, Care and Treatment in the U.S.: A 30 Year Comparative Analysis. National Organization for Rare Disorders; 2020 Nov [cited 2023 Feb 8]. Available from: http://rarediseases.org/wp content/uploads/2020/11/nrd - 2088障碍- 30 -年-调查- report_fnl - 2.pdf

Veeva ID: Z4-62042
Date of preparation: February 2024