🚨 IT’S RARE DISEASE DAY! 🚨 For #RareDiseaseDay 2024 we're highlighting key facts and figures to educate change makers on the shared challenges of our community. Alongside these we will share the #StoriesBehindTheStats. 👉 ow.ly/4j7E50QIXEY

Today we raise awareness and ignite change for the 300 million individuals worldwide living with a #raredisease. One of these diseases is r(20), which falls into the 5% or >6,000 ultra-rare conditions. Pls share this post… Let's spread awareness together. 💜 #RareDiseaseDay🧬

Our favourite day of the year is here - Rare Disease Day! Join us and the rare disease community around the world to spread knowledge about rare diseases All our articles are free to read & have a plain language summary to make them more accessible to patients #RareDiseaseDay

The long-term cause of the Rare Disease Day campaign is to achieve equitable access to diagnosis, treatment, health and social care and social opportunity for people affected by a rare disease. Rare is Strong! Proud! Many! #RareDiseaseDay2024

📣 Call for Papers Launching today, a new collection of articles which seeks to bring together experts in the field of rare infectious diseases 🔓#OpenAccess 🤝 Collaboration with Therapeutic Advances in Infectious Disease 📅 Deadline 30 Sept 🔗 bit.ly/3V55wV4 Sage Clinical Med Tiina Urv #RareDiseaseDay

Why is Gene People involved in #RareDiseaseDay ? Over 70% of all rare diseases are genetic. We want to raise awareness of these and all rare diseases this special day.

Here at Therapeutic Advances in Rare Disease we are committed to making our research as accessible as possible for the rare disease community. See the message below from our Editor in Chief Tiina Urv All our articles are published open access, so free to read all year not just on #RareDiseaseDay!

The visible challenges facing those living with rare diseases are the tip of the iceberg. Let’s not settle for skimming the surface. It's time to #ActRare2024, dive deep, and emerge with solutions for a brighter future. 🏊 Dive into our policy demands: eurordis.org/actrare2024/

📣 New online! A United States-based patient-reported adult polyglucosan body disease registry: initial results 🔗 bit.ly/3TYfk1i 🔓 Open Access Sage Journals #OpenAccess #FOAMed #PolyglucosanBodyDisease #RareDisease

Mental health matters! 🧠 At #ECRD2024, we will be co-creating a new toolkit for the rare disease community by exploring best practices and tools that can be drawn on and scaled to form the basis of the new Toolkit. Less than one month to go! Join us 👉 go.eurordis.org/hVPVPA

It's finally arrived: April 17th! Today marks #WorldHemophiliaDay—a day dedicated to celebrating our global community. From all of us at the WFH, we wish you a joyous #WHD2024! 🎉

Today is #WorldHemophiliaDay! 🩸 So we wanted to share some of the research we have published in this area! Improving patient informed consent for #haemophilia #genetherapy: the case for change bit.ly/49FONvh Laurence Woollard Dr Rich Gorman Dakota Rosenfelt, PharmD, RPh, MBA Sage Clinical Med

Sharing more research from Therapeutic Advances in Rare Disease on #WorldHemophiliaDay! 🩸 Etranacogene dezaparvovec for hemophilia B gene therapy bit.ly/445jo4h Courtney Thornburg Sage Clinical Med #hemophiliaB #hemophilia #RareDisease #FOAMed #TARareDisease #OpenAccess

Less than three weeks remain until the remarkable MENA Congress for Rare Diseases occurs. 🗓 Save The Date: May 16-19, 2024 🏨 Venue: Beach Rotana Hotel, Abu Dhabi, UAE Make sure not miss out this phenomenal chance! Register now at menararediseases.com #menararediseases2024

Each year, around 6,000 children are born in the UK with a genetic condition that will likely remain undiagnosed. Today on Undiagnosed Children's Day, we're highlighting SWAN UK (syndromes without a name), a support community for families affected by a syndrome without a name. ow.ly/wgKu50RfYMa

📣Out now! Developing a pathway to clinical trials for #CACNA1A-related epilepsies: A patient organization perspective 🔗 bit.ly/3Wqk4j4 Part of our Roadmaps and Resources to Advance Therapy Development for Rare Epilepsies collection Sage Journals Tiina Urv CACNA1A Foundation

Thrilled to announce our collaboration with Therapeutic Advances in Rare Disease for a Special Collection on Rare Infectious Diseases! 🌐📚 Submit your research by Sep 30, 2024. Details: bit.ly/3uHbuRD

We are delighted to announce that Therapeutic Advances in Rare Disease has published the submitted abstracts. View the published abstract: doi.org/10.1177/263300… For more details and registration: menararediseases.com

We are excited to announce that we will be hosting the first ever REMEDi4ALL Repurposing Bootcamp! Held back-to-back with #OpenAcademy2025, this is a specialised training programme designed to empower you to drive innovation through drug repurposing. ➡️ go.eurordis.org/utugS3