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Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community. GL/NP/0116.

We are driven by a mission to change what it means to live with a rare disease.

20/10/2026

Imagine knowing something wasn't right, but no one could tell you why.

That was Lisa's reality for years. From serious dental issues in childhood to muscle pain that made everyday activities, even just lifting her arms, a struggle, she knew her symptoms weren't normal. But finding answers wasn't easy.

Lisa kept asking questions, trusted her instincts and continued to stand up for herself. After decades of searching, she finally had an answer: (HPP), a disease with a wide range of symptoms often overlooked due to how rare it is.

Today, she's sharing her story in hopes that others facing unanswered symptoms feel seen, heard and less alone.

Watch Rare Connections in HPP to hear how Lisa and others are navigating life with this :

15/10/2026

"Giving up is not an option."

Those words became a motto for Antje, who has lived with overwhelming fatigue and severe muscle and bone pain for decades before learning the cause: (HPP), a with a broad range of symptoms that can make it difficult to diagnose.

In Rare Connections in HPP, Antje reflects on how this disease has shaped her childhood and continued to impact her life today and how her deep inner strength, together with the unwavering support of loved ones, has kept her moving forward.

Hear her story and others in Rare Connections in HPP:

11/10/2026

Three people. Three countries. One diagnosis.

Today in Boston, we premiered Rare Connections in HPP at , a new short film putting a spotlight on (HPP), a rare disease that is often overlooked and misunderstood.

Through the lived experiences of Lisa, Mario and Antje – three people navigating life with HPP from around the world – the film shows how a can shape a person's life yet remain unrecognized for decades.

Step into their shoes and discover the connections that unite them:

10/10/2026

For people living with (HPP), a correct diagnosis does not always come easy.

Behind years, sometimes a decade, of waiting, are constant uncertainty, numerous specialist visits and the emotional weight of not having a name to put to the signs or explain them to others.

Earlier and accurate diagnosis has the potential to change what those years hold for people living with this rare metabolic disease.

30/09/2026

Generalized (gMG) can affect an entire household, not just the individual living with this rare neurological disease.

84% of people living with gMG receive care from nonprofessional care partners, such as friends or family, to help navigate everyday tasks, such as brushing teeth or helping with chores and errands.

The need for such support reflects not only the depth of the disease burden but the far-reaching effects gMG can have on daily life.

24/09/2026

Today is Global aHUS Awareness Day. Atypical hemolytic uremic syndrome (aHUS) is a rare disease which can present with potentially severe complications. This week, our team is honored to attend the aHUS Foundation Summit, Sept. 24–27, to listen, learn and connect with patients, caregivers and advocates in the aHUS community.

23/09/2026

PNH Awareness Week is a time to recognize the people and families affected by paroxysmal nocturnal hemoglobinuria, a rare disease caused by an acquired genetic mutation (not inherited) that may happen any time after birth.

At Alexion, AstraZeneca Rare Disease, we stand with the patients, caregivers, advocates and clinicians working to bring greater visibility to the PNH community—every day and throughout PNH Awareness Week.

15/09/2026

September marks Newborn Screening Awareness Month — an opportunity to recognize the role early identification can play in helping children and families affected by rare disease receive answers sooner. For many families, the path to diagnosis can be long and uncertain. At Alexion, AstraZeneca Rare Disease, we are committed to supporting efforts that improve the diagnostic experience for people living with rare diseases and to recognizing those working to expand awareness of newborn screening.

https://alexion.us/news-centre-resources/articles/advancing-newborn-screening-through-earlier-access-to-diagnostic-testing

07/09/2026

This Labor Day, we recognize the researchers, clinicians, care partners, advocates and colleagues working on behalf of the rare disease community. Thank you for the dedication you bring to this work and for the impact you have on patients, families and communities. Wishing everyone a safe and restful holiday from Alexion, AstraZeneca Rare Disease.

19/08/2026

National Patient Advocacy Day is an opportunity to recognize the voices and perspectives that help move the rare disease community forward. At Alexion, AstraZeneca Rare Disease, advocacy is part of our everyday commitment. We listen to and partner with patients, caregivers and advocacy organizations to better understand their experiences, support meaningful collaboration and help shape initiatives that address the needs of the communities we serve.

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