Alexion Pharmaceuticals, Inc.

While individually rare, these diseases collectively affect millions across Europe, presenting significant challenges for patients and their families. The comprehensive study from Charles River Associates dives deep into the economic and emotional burdens faced by those living with rare diseases. Join us in raising awareness and advocating for meaningful change by exploring the findings and becoming part of the solution: http://spr.ly/6048IjLXs #RareDisease

Over 400 million people worldwide live with a #RareDisease. But did you know 1 of 2 patients diagnosed with a rare disease is a child?

Amyloidosis is a disease where diagnosis and treatment are often delayed because it can mimic signs and symptoms of other diseases. Learn more about our work to address amyloidosis, often called “the great pretender.” http://spr.ly/6045IyZqv

“The potential for our research to help address their unmet needs, such as receiving a timely diagnosis and treatment, motivates me every day.” -Cristina Quarta on our work to address the unmet needs for people living with amyloidosis http://spr.ly/6043IueMV

We spoke with Seng H. Cheng, Head of Research and Product Development about what creativity, serial innovation and passion looks like in rare disease R&D. Watch the video to learn more.

Les équipes d’Alexion France participent aux Journées de la Société Francophone du Nerf Périphérique #SFNP #SFENMG qui se dérouleront au Newcap Event Center à Paris, du 31 janvier au 1er février 2025. Cet évènement est l’occasion d’évoquer les spécificités liées aux maladies rares neurologiques, et d’échanger avec les acteurs de santé. Au plaisir d’échanger avec vous sur notre stand ! #maladiesrares #alexion Avec Cécile Auriol, Julie Deléglise, Stephanie Thomas, Justine Paret , Amina Kallel , Anne-Laure ALEC , Vincent Pinton , Karim Mhamdi , Sonia YACOUB , Marina Iskandar , Agnès Denarnaud, Jean-Christophe Delain, Walid BEGHDADI, PhD Clémence d'Aietti, Yannick Bondabou Pascal Bernardinello Ce contenu est destiné uniquement aux résidents de France et de Belgique.

Designing a #RareDisease #ClinicalTrial first requires a deep understanding of the unique challenges in this space—like a limited understanding of the disease and that few diagnostic tools are available. In this video, learn about the new approaches we continue to pioneer to accelerate the discovery and development of transformative medicines.

Why is conducting #ClinicalTrials for rare diseases difficult? Because studying a #RareDisease poses unique challenges and requires outside-of-the-box thinking. Watch the video to learn why the need for transformative medicines is great for those living with rare diseases.

New research from AstraZeneca in Nature Portfolio demonstrates the transformative potential for gene editing to transform outcomes for people living with rare genetic disorders by addressing their underlying causes. The new gene editing tool showed enhanced precision to correct genetic mutations, while reducing off-target effects. This advancement could help accelerate potential new treatments for a range of diseases, including rare conditions—of which 80% are believed to have a genetic cause. Learn more here: http://spr.ly/6045aZ7vf

Hear from our diagnostics team, Banu Sankaran, Tom Defay and Chathuri Daluwatte, PhD, about promising areas of diagnostic innovation that aim to shorten the path to diagnosis. By harnessing these technologies for diagnostics, we are working to bring new hope to people impacted by rare diseases.