罕见的疾病

改变罕见病患者的生活

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澳门第一赌城在线娱乐的承诺

Our mission is to transform the lives of people affected by rare diseases By 理解ing patients’ unique needs, 澳门第一赌城在线娱乐可以研究和开发创新药物, 支持获取和倡导罕见病社区.
 

澳门第一赌城在线娱乐认为倾听是澳门第一赌城在线娱乐的责任, 理解, and change the lives of patients and those who work tirelessly to help them. Our innovation begins with 理解ing people living with rare diseases, 是什么推动了澳门第一赌城在线娱乐所有的努力.

罕见疾病的数量

4亿年

世界各地的人们都患有一种罕见的疾病1

10,000

rare diseases are known to exist today, but more than 90% do not have an approved treatment option2,3

5 +年

to accurate diagnosis, a path that includes multiple doctors, specialists and misdiagnoses4

澳门第一赌城在线娱乐在罕见病方面的策略

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. 今天, 作为澳门在线赌城娱乐的一部分, we are building bridges across our scientific platforms with a focus on bringing more innovative medicines to people worldwide.

澳门第一赌城在线娱乐的罕见病战略侧重于三个核心优先事项:

1. Accelerate by creating smart and efficient strategies to speed access to our medicines for patients.

2. 通过科学投资进行创新, 平台和功能, 包括使用澳门第一赌城在线娱乐自己的技术和研究能力.

3. Reach beyond our current geographic footprint to as many rare disease patients as possible.

罕见疾病新闻和故事

罕见病临床试验创新
推动罕见病社区的卫生公平

澳门第一赌城在线娱乐的R&D方法

30年来, Alexion’s pioneering science has led to the development of transformational medicines for rare diseases, 今天，澳门第一赌城在线娱乐继续引领这一潮流. Our success is tied to listening, to 理解ing, and responding to the communities we serve.

罕见病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 理解ing of many rare conditions and the needs of the people living with them.

澳门第一赌城在线娱乐的人民

We invest in and value people who believe in the importance of our purpose and 理解 what it takes to deliver on it. 澳门第一赌城在线娱乐所做的一切, 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. 澳门第一赌城在线娱乐的文化植根于诚信, 包容, 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.

马克•Dunoyer Chief Executive Officer, Alexion and Chief Strategy Officer, AstraZeneca
丽莎希

副总裁，美国业务部门领导神经病学

查看Lisa的个人资料
朱迪Campagnari

执行董事、联盟及整合小组(AIT)主管

查看Judy的个人资料

加入澳门第一赌城在线娱乐的团队

澳门第一赌城在线娱乐是创新者、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.

Alexion求职网站

澳门第一赌城在线娱乐的药品

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

澳门第一赌城在线娱乐的药品 are approved in individual countries for specific uses and the 信息rmation we provide for patients is governed by local regulations. 在某些情况下, 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, 信息rmation 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.

在AZ网络中找到您的国家/地区网站
澳门第一赌城在线娱乐的全部药品组合

澳门第一赌城在线娱乐的罕见病产品线

每天都在推进澳门第一赌城在线娱乐的管道

We’re building on our fundamental 理解ing of complement biology and advancing on our core therapy areas of haematology, 肾脏学, 神经学, 代谢, 和心脏病学. 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 III/LCM Projects: refers to assets that are pivotal in Phase II/III, 或者已经提交监管部门批准的, and may include assets that are now launched in one or more major markets (removed when launched in all applicable major markets).

澳门第一赌城在线娱乐的罕见病产品线

参考文献

1. 罕见病常识[互联网]. 2024年[引自2024年1月17日]. 可从:http://globalgenes获得.org/rare-disease-facts/

2. Fermaglich LJ, Miller KL. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the Forty Years of the Orphan Drug Act. 罕见病孤儿杂志. 2023 6月23日;18(1). doi: 10.1186/s13023-023-02790-7

3. 约[网络]. U.S. Department of Health and Human Services; [cited 2024 Jan 17]. 可从:http://rarediseases获得.信息.国家卫生研究院.gov /对

4. 美国罕见病诊断、护理和治疗的障碍.S.: 30年的比较分析. National Organization for Rare Disorders; 2020 Nov [cited 2023 Feb 8]. 可从:http://rarediseases获得.org/wp-content/uploads/2020/11/NRD-2088-Barriers-30-Yr-Survey-Report_FNL-2.pdf

Veeva ID: Z4-62042
筹备日期:2024年2月