🔥The 9th Round of Easy Loan, Earn $40 Reward is in progress❗️ ⏰ Promotion Period: January 15th - Feburary 15th, 2025 👉 Register now and check more details at gate.io/campaigns/358

🎓 Are you a recent graduate with an interest in rare diseases, neurology or biotech? We have a very limited number of FREE places available to attend the TREAT-NMD Duchenne Muscular Dystrophy Industry Day for recent graduates or interns in the field. 🧬 Learn from

🎉 The Becker Muscular Dystrophy Education & Engagement Days are back this September! Join us for two incredible events designed for individuals aged 14+ living with Becker muscular dystrophy and their families. 📍 Newcastle, UK – 5–6 September 2025 (For attendees from the UK,

🔬 The IRDiRC Diagnostics Scientific Committee is recruiting 2 new members! 🌍 Seeking diagnostic lab scientists from North America, North/Central Asia, or Australasia. 📅 Apply by 23 July 2025 📧 scientific.secretariat[at]irdirc.org 🔗 irdirc.org/dsc/

⏳ Just Over a Week Left to Register - time is running out! DMD Industry Day is happening on 11th July in Prague, and there’s just over a week left to secure your place. 💡 Why Attend? ✅ Gain expert insights from key opinion leaders ✅ Gain a comprehensive overview of DMD ✅

Are you a student in medicine, life sciences, or a related field? Join us for the TREAT-NMD Duchenne Muscular Dystrophy Industry Day – a free, expert-led symposium on the latest in DMD research, treatment, and care. 📍 Grandior Hotel, Prague 🗓️ Friday 11 July 2025 | 09:00–18:30

⏳ Just Over a Week Left to Register! Time is running out! DMD Industry Day is happening on 11th July in Prague, and there’s just over a week left to secure your place. 💡 Why Attend? ✅ Gain expert insights from key opinion leaders ✅ Gain a comprehensive overview of DMD ✅

📢 Exciting news! Dr. Helen Malherbe is the new Vice Chair of the Interdisciplinary Scientific Committee (ISC)! 🌟 Her appointment brings valuable expertise and vision to the ISC, where she will help shape strategic direction. 🔗 irdirc.org/isc/ #ISC #IRDiRC

As we look ahead to next week’s DMD & SMA Educational Masterclasses in Prague, we are proud to recognise our Platinum Sponsor, Roche, for their outstanding support of both programmes. Roche’s commitment to advancing education and innovation in neuromuscular care plays a pivotal

With UK Dementia Research Institute, we're funding a first-in-class gene therapy targeting a root cause of motor neuron disease: gene errors caused by TDP-43 dysfunction. This new treatment aims to correct faulty gene messages and deliver long-lasting treatment in a single dose.

We're calling on government, regulators, industry, academia, the NHS and patient groups to act together and #ChangeTheRareFuture for people living with rare conditions in the UK. Read the report here - lifearc.org/rd-report/ Genetic Alliance UK

This morning, we've gathered with over 100 policy makers, charity, academic, clinical and industry partners, and patient experts to talk about our new report with Genetic Alliance UK, and how we can accelerate rare disease research and development in the UK. #ChangeTheRareFuture

Our Rare Disease Chair, Amit Nathwani, shares why it's so important that we #ChangeTheRareFuture for the nearly 3.5 million people with rare diseases in the UK at our report launch in Westminster. Genetic Alliance UK

As we gear up for next week's DMD & SMA Educational Masterclass in Prague, we would like to extend a huge thank you to our gold sponsor, Biogen, for their generous support of the SMA programme. Your partnership helps us share vital knowledge with healthcare professionals around

"This Government remains committed to improving the lives of people with rare diseases… This is not something that [we] can do alone. We will do it in partnership with experts and with people with rare diseases and their families." Ashley Dalton MP #ChangeTheRareFuture

"The future of medicine – for all of us – is more personalised, more precise, and more patient-led... We must act today to work together and to transform the system for the millions of people living with rare diseases." -Sam Barrell, LifeArc CEO #ChangeTheRareFuture

More rare disease R&D in the UK means faster diagnoses, better treatments and hope for millions living with rare conditions. We are proud to collaborate with @LifeArc and task force members on this, read recommendations here: t.ly/8lqIH Let’s #ChangeTheRareFuture

Last chance to make your voice heard!🗣️ #ERDERA has launched a survey to explore how #RareDisease patients can contribute to EU-funded research. 🔐 Confidential & 🤝 Co-created with patients 🔗 Take the survey: shorturl.at/ePVuL 📄 More: shorturl.at/c03db EURORDIS-Rare Diseases Europe

🚨 Research Grant Opportunity! The WSS Foundation & the Orphan Disease Center are offering 3⃣ two-year grants ($90K each) for #WiedemannSteinerSyndrome research: 📅 LOIs due Aug 8, 2025 🔗 Apply now: orphandiseasecenter.squarespace.com/grants/wiedema…

🌧️ Good morning from a rainy Prague! We’re excited to welcome healthcare professionals from across the globe to the TREAT-NMD® Spinal Muscular Atrophy (SMA) Masterclass, starting today. This accredited, expert-led programme offers an interactive learning experience for

🌟 Join the 2025 RARE Drug Development Symposium! 🌟 📅 Sept 3–4, Boston, MA, USA A hands-on, expert-led event empowering #RareDisease advocates to drive progress in drug development. 💊🔬 👉 Learn more & register: globalgenes.org/rare-drug-deve… Global Genes #RareDrugDev25