Our Mitochondrial and inhertited metabolic disease taskforce led by Critical Path Institute (C-Path) now has a mission statement accepted by the members! Our mission is: ☑ Standardizing and integrating mitochondrial and inherited metabolic diseases data into the RDCA-DAP®. ☑ Optimizing clinical trial design and endpoints. ☑ Enhancing communication and collaboration among stakeholders, and the medical and research communities at large, all aimed at improving the lives of those affected by these diseases. #mitochondrialdisease #leighsyndrome
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Cold agglutinin disease (CAD) is a subtype of autoimmune hemolytic anemia (AIHA) where individuals experience complex symptoms triggered by exposure to cold. Improving treatment outcomes and reducing symptom burden through consensus recommendations is crucial. Join us as Catherine M. Broome, MD, Professor of Medicine at Georgetown University School of Medicine, shares her expert insights on: ⚪ Clinical and laboratory features for timely diagnosis ⚪ Emerging data on novel complement inhibitors targeting the classical complement pathway ⚪ Supportive care strategies for managing anemia, cold-induced circulatory symptoms, and treatment-related toxicities ⚪ And much more! Register for this insightful live #CME webinar here: https://bit.ly/3Y6Yz7t #ColdAgglutininDisease #AnemiaManagement #MedicalWebinar #ExpertInsights #HealthcareProfessionals
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DualDur is the fruit of 40 years of scientific work, tens of thousands of tests performed and confirmed by the largest Lyme diagnostic clinical research in Europe. The European Parliament resolution on Lyme disease proposes DualDur and similar innovative approaches for the early detection of the disease. Learn more about DualDur on this link: https://lnkd.in/gwuqvzH #lyme #borrelia #lymedisease #lymediagnostics
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Rare Disease Clinical Trials vs. Standard Trials: What You Need to Know🔍 Clinical trials aren’t one-size-fits-all. Rare diseases demand unique strategies and innovative approaches. 🚀 From recruitment challenges to market dynamics, these trials shape the future of medicine in powerful ways. 🌱 Curious about the key differences and best practices? Explore how understanding these trials can revolutionize research and lead to groundbreaking treatments. 💡 Read the full article to get the inside scoop! https://lnkd.in/gkJezfdW #ClinicalTrials #RareDiseases #MedicalResearch #HealthcareInnovation
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#MetX, funded by the Metrodora Foundation and in partnership with Metrodora Institute and PrecisionLife, is a first-of-its-kind study to create more accurate diagnostics and precision therapies for patients living with complex multisystem disorders. The first participants have begun receiving their #WGS results in detailed research reports with PrecisionLife insights that identify the possible underlying mechanisms of their disease - a significant breakthrough in #PrecisionMedicine for complex diseases. Read more in the Metrodora Foundation newsletter ft. Amy Rochlin and Steve Gardner. ➡️ https://lnkd.in/ebgrcAve
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According to the National Organization for Rare Disorders there are more than 7,000 different rare diseases, of which 95% lack any FDA-approved treatment. From uncovering genetic factors to exploring potential therapies, collaboration among researchers, healthcare professionals, and advocacy groups is vital to advance clinical research and potential treatments. As we approach #RareDiseaseDay, we join the global rare disease community in advocating for increased research and collaboration. Together, we can amplify awareness, accelerate discoveries, and pave the way for innovative treatments, offering hope for the 300 million people worldwide living with a #raredisease. Learn more about Rare Disease Day: https://lnkd.in/gk6hbAF
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Rare Disease Clinical Trials vs. Standard Trials: What You Need to Know🔍 Clinical trials aren’t one-size-fits-all. Rare diseases demand unique strategies and innovative approaches. 🚀 From recruitment challenges to market dynamics, these trials shape the future of medicine in powerful ways. 🌱 Curious about the key differences and best practices? Explore how understanding these trials can revolutionize research and lead to groundbreaking treatments. 💡 Read the full article to get the inside scoop! https://lnkd.in/gwewBsn3 #ClinicalTrials #RareDiseases #MedicalResearch
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Yesterday, the world observed #RareDiseaseDay, a time to spotlight the challenges faced by those with uncommon medical conditions. Were you aware that 72% of rare diseases are genetic, and nearly 1 out of 5 cancers falls into this category? This statistic, sourced from www.rarediseaseday.org, Is a reminder of the resilience of individuals battling these diseases and a call to action for increased awareness, research, and support. Let's stand together to ensure no one facing a rare disease feels alone, advocating for progress in healthcare and fostering inclusivity. To learn more and get involved, visit: https://bit.ly/3CYMXbp
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Rare Disease Clinical Trials vs. Standard Trials: What You Need to Know🔍 Clinical trials aren’t one-size-fits-all. Rare diseases demand unique strategies and innovative approaches. 🚀 From recruitment challenges to market dynamics, these trials shape the future of medicine in powerful ways. 🌱 Curious about the key differences and best practices? Explore how understanding these trials can revolutionize research and lead to groundbreaking treatments. 💡 Read the full article to get the inside scoop! https://lnkd.in/g6rr-Gq7 #ClinicalTrials #RareDiseases #MedicalResearch #HealthcareInnovation
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Today is national clinical trials day, but it’s disappointing that it’s still difficult to recruit for rare disease trials because only a tiny minority of diseases have access to full molecular characterisation and registry. It’s clear when you have a molecularly characterised cohort who are accessible via a registry, ready for clinical trials, there is the potential for a huge return on investment- just look at CF. We need this across rare disease, so that therapies can be developed for the 95% who don’t have approved therapies. Today was my first visit to the House of Lords to discuss the solutions. #raredisease #rarediseases #clinicaltrial #clinicaltrialsday #clinicaltrials
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Rare Disease Clinical Trials vs. Standard Trials: What You Need to Know🔍 Clinical trials aren’t one-size-fits-all. Rare diseases demand unique strategies and innovative approaches. 🚀 From recruitment challenges to market dynamics, these trials shape the future of medicine in powerful ways. 🌱 Curious about the key differences and best practices? Explore how understanding these trials can revolutionize research and lead to groundbreaking treatments. 💡 Read the full article to get the inside scoop! https://lnkd.in/g9sPuKpb #ClinicalTrials #RareDiseases #MedicalResearch #HealthcareInnovation
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