Indo US Organization for Rare Diseases (IndoUSrare)’s Post

SPHERE's Executive Director, Professor Chris White, was recently invited to attend the Crohn's Colitis Cure annual meeting where progress toward establishing a community-led cloud-based medical record for its 15000+ consumers with inflammatory bowel disease was presented. This collaboration represents a significant step forward in enhancing specialist engaged care to patients with Inflammatory Bowel Disease (#IBD) wherever they are across the globe, while providing a robust resource of patient #realworddata and insights. In the complex world of IBD care this kind of comprehensive data via public participation is a game-changer. It opens new avenues for collaborative research, implementing and identifying effective therapies and management —and ultimately, a cure. We are excited to see how this integration will propel Crohn's Colitis Cure's mission and contribute to transforming the lives of those affected by IBD. More importantly, SPHERE recognises the value of integrating Crohn's Colitis Cure's data with hospital-based records and other models of community led and specialist engaged models that could follow this paradigm. Joseph P. Health Translation SA Georgina Hold Bill Petch Professor Jane M Andrews Susan Connor #crohns #crohnscolitiscure #inflammatoryboweldisease

Conducting clinical studies in rare diseases presents a unique set of challenges. Success can only be achieved if the burden on both sites and patients is minimized while ensuring reliable data collection. In this article, Juliet (Jules) Moritz, Senior Vice President, Strategic Solutions and Patient Centricity, ERGOMED Clinical Research, provides insight into why Ergomed is ideally positioned to design and conduct rare disease clinical trials with minimal unexpected difficulties. Find out how we ease the burden of Rare Disease research: https://lnkd.in/eMjBQFtU #RareDisease #ErgomedCRO #HealthcareInnovation #ClinicalResearch

At the heart of Alzheimer’s disease research is the patient experience. C-Path emphasizes patient-centered research to ensure that treatments and trials align with what matters most to those living with the disease. By incorporating patient feedback into tools to support trial designs, C-Path aims to accelerate development of therapies that not only target the disease but improve quality of life. Patient voices are important as they shape the trajectory of Alzheimer’s disease research and help prioritize what needs the most attention. It’s vital to ensure that patients’ voices and their wellbeing aren’t forgotten in scientific progress. Learn more about how C-Path is driving these efforts by visiting c-path.org/cpad. #CPath #AlzheimersDiseaseAwarenessMonth #CPAD #Biomarkers #PatientVoice

News from our collaborators! From Bateman Horne Center of Excellence: At the Bateman Horne Center, we’re committed to advancing treatments for chronic illnesses. Our latest blog highlights promising results from two clinical trials: 🔬 RESTORE ME Trial: 📉 Fatigue reduced by 27% with oxaloacetate—a quality of life improvement for ME/CFS patients! 💊 Long COVID Trial: Low-dose valacyclovir + celecoxib showed meaningful fatigue reduction for Long COVID sufferers. 📖 Read the blog from BHC's Director of Research, Suzanne Vernon, PhD, here: https://loom.ly/YVzZ95A #ClinicalTrials #MECFS #LongCOVID

At the heart of Alzheimer’s disease research is the patient experience. C-Path emphasizes patient-centered research to ensure that treatments and trials align with what matters most to those living with the disease. By incorporating patient feedback into tools to support trial designs, C-Path aims to accelerate development of therapies that not only target the disease but improve quality of life. Patient voices are important as they shape the trajectory of Alzheimer’s disease research and help prioritize what needs the most attention. It’s vital to ensure that patients’ voices and their wellbeing aren’t forgotten in scientific progress. Learn more about how C-Path is driving these efforts by visiting c-path.org/cpad. #CPath #AlzheimersDiseaseAwarenessMonth #CPAD #Biomarkers #PatientVoice

We sit down with Kim Moran, SVP & Head of U.S. Rare Diseases at UCB, to discuss the vital role of patient collaboration in driving innovation for the care and support of Myasthenia Gravis (MG) patients. Inspired by patients and driven by science, UCB is dedicated to exploring the patient journey to uncover uncertainties and develop new advancements for MG patients. Watch the full story on TheBalancingAct.com/rare #BehindTheMystery #MyastheniaGravis #MyastheniaGravisAwareness #MGAwareness #RareDisease #UCB #RareDiseaseAwareness

We sit down with Kim Moran, SVP & Head of U.S. Rare Diseases at UCB, to discuss the vital role of patient collaboration in driving innovation for the care and support of Myasthenia Gravis (MG) patients. Inspired by patients and driven by science, UCB is dedicated to exploring the patient journey to uncover uncertainties and develop new advancements for MG patients. Watch the full story on TheBalancingAct.com/rare #BehindTheMystery #MyastheniaGravis #MyastheniaGravisAwareness #MGAwareness #RareDisease #UCB #RareDiseaseAwareness

For US #HCPs at #EHA2024: don’t miss Dr. Wilfried Roeloffzen present efficacy & safety results from Cohort D of the Phase 2 CARTITUDE-2 study, investigating a BCMA CAR-T therapy in patients with newly diagnosed #MultipleMyeloma & suboptimal response to frontline ASCT. Click here to see the presentation: https://lnkd.in/e2aJqRtM

Approximately 56.9 million people worldwide are living with #Alzheimer’s Disease and other dementias, projected to rise to 78 million by 2030. With few approved and effective therapeutic interventions available, there is an urgent need for new treatment options to address the profound impact on patients, families, and society. Download this article to explore the challenges of AD and #neurodegenerative clinical trials, recent successes in the field, and strategies for achieving success in these trials: https://lnkd.in/gHevTguM

In July 2023, Leqembi® became the first disease-modifying treatment to receive traditional approval from the FDA. Making this new therapy available has required health systems to pivot and collaborate to ensure access and safety. On June 27, the webinar “A new era of treatment: Lessons in anti-amyloid care pathways” will review available treatments and therapies for Alzheimer's disease, key considerations for ensuring safe access to new therapies and nonpharmacologic strategies for caring for and treating Alzheimer's and related dementias. It’s presented in partnership with HANYS and New York Presbyterian/Columbia University. https://bit.ly/4bDVr6U