Allen Moradian’s Post

Looking for an innovative approach to rare disease clinical trials, demonstrating substantial sample size reduction? 🚀 Discover our groundbreaking research featured in the Orphanet Journal of Rare Diseases, where we unveil how the utilization of multiple outcomes in a clinical trial design for Mucopolysaccharidosis type IIIA (MPS IIIA) has yielded remarkable value . This innovative methodology has resulted in a significant decrease in the required sample size, ensuring adequate statistical power to detect meaningful differences in targeted outcomes. As a result, the study not only became feasible but also represented a pivotal advancement in the quest to develop treatments for rare diseases such as MPS IIIA, characterized by severely limited patient populations. Unlock the comprehensive insights and potential implications of this research by accessing the full paper 📖 : https://lnkd.in/e9NfBNru For more information or inquiries, please contact us at info@one2treat.com #ClinicalTrials #MPSIIIA #PatientCentricity #ReducedSampleSize #InnovativeResearch

You have A LOT of challenges in the efficient conduct of your #clinicaltrials. The #one2treat solution may be able to address your #raredisease needs. Read below to see how this innovative methodology permitted a significant DECREASE in the required sample size WITHOUT compromising adequate statistical power to detect meaningful differences in targeted outcomes! #MPSIIIA #ReducedSampleSize #InnovativeStatistics #ClinicalTrialOptimization

All articles in the Journal of Clinical Medicine (JCM) MDPI (ISSN: 2077-0383) Vol 13, Issue 14, are now freely available to access, read and download: https://brnw.ch/21wLuzn COVER STORY: There is a well-established relationship between different subsets of idiopathic inflammatory myopathies (IIMs, myositis) and interstitial lung disease (ILD), with lung complications sometimes presenting prior to myopathic manifestations. The subtypes of myositis include those that are strongly associated with ILD. Research from Temple Health – Temple University Health System has shown that in certain patients, these can then be further divided into subtypes using myositis-specific antibodies (MSAs), which are specific for myositis, and myositis-associated antibodies (MAAs), which can be found in myositis in overlap syndromes with other connective tissue diseases (CTDs). The aim of this review is to highlight the clinical features, diagnostic criteria, important histopathologic, laboratory, and radiographic features, and treatment modalities for those patients with myositis-associated ILD. Read more: https://brnw.ch/21wLuzn #mdpi #openaccess #myositis #lungdisease #research

"A Review of Myositis-Associated Interstitial Lung Disease" by Renuka Kannappan, M.D. et al Temple University Hospital Welcome to read here: mdpi.com/2865810 ✅ This Review is included in Special Issue "Clinical Advances in Interstitial Lung Diseases", it is still open and we welcome more submissions! 💌 Submission link: mdpi.com/si/185699

What does the future hold for rare disease treatment? 🔬    Access to information online will help expedite disease diagnosis further.     Online Mendelian Inheritance in Man (OMIM)17 and Orphanet18 are two highly reliable databases that collect information on newly reported diseases and disease genes.     Access to more information about patients, their symptoms and progress will likely be found from the new sources like surveys of specialty physicians, clinical labs, and patient organizations, or from rare disease networks.      People like you are the focal point of clinical research and drug development. Companies and researchers have come to rely on feedback from patients to better understand what it’s like to live with a disease, and that information can help them in the development of clinical studies.     That means that your contributions and participation are critical to the development of new treatments for others like you with a rare disease. 💙    -    #raredisease #medicalresearch #clinicaltrials #clinicaltrial #clinicalstudy #rarediseaseawareness #rarediseases #KnowRare

#HighlyCitedPapers 📝Urinary Galectin-3 as a Novel Biomarker for the Prediction of Renal Fibrosis and Kidney Disease Progression — Ou et al. In this paper, the authors aimed to examine whether the urinary Gal-3 level is also a useful biomarker enabling the accurate prediction of the risk of #kidney disease progression. Full text is available 👇 https://lnkd.in/dM_HJsZN #medicine #health #research #science

🙌Recommended Paper "A Review of Myositis-Associated Interstitial Lung Disease" by Renuka Kannappan, M.D. et al @Temple University Hospital 🧐 Read here: https://lnkd.in/g7XYT9QV 👉 This article belongs to the Special Issue "Clinical Advances in Interstitial Lung Diseases", it is still open and we welcome more submissions! 💌 Submission link: mdpi.com/si/185699 #InterstitialLungDisease #ILD Journal of Clinical Medicine (JCM) MDPI

CAMRIS works to improve patient outcomes and advance health through discovery and innovation by: -Developing efficacious diagnostics and therapies to prevent and treat diseases. -Investigating the biological mechanisms that contribute to disease states. -Implementing Good Laboratory Practice (GLP) standards to ensure reliable and accurate laboratory testing. -Understanding transmission, pathogenesis, and host immune responses. Please read the below info sheet to learn about our Laboratory Services’ capabilities and commitment to the broader field of clinical research

🌟 New Research Alert! 🌟 📢 We are excited to announce the publication of a groundbreaking study on Evusheld Efficacy and Safety in Autoimmune Rheumatic Diseases Treated by Rituximab: A Prospective Observational Trial! Research paper by Lisa Kaly, Mark Volevich, Shira Keret, Aniela Shoval, Dr. Itzhak Rosner, Michael Rozenbaum, Nina Boulman, Emilia Hardak, Gleb Slobodin, Doron Rimar, Bnai Zion Medical Center, Technion - Israel Institute of Technology 🔍 Keywords: Evusheld, COVID-19 infection, B-cell depletion therapy, autoimmune rheumatic disease This study dives deep into the impact of Evusheld for patients undergoing B-cell depletion therapy with Rituximab, offering critical insights into: ✅ The protection it provides against COVID-19 ✅ Safety considerations for autoimmune rheumatic disease patients ✅ Its real-world implications in managing immunocompromised individuals Explore this timely research, especially relevant in a post-pandemic world, and discover how Evusheld may transform treatment strategies for vulnerable populations! Read the full article here: https://lnkd.in/g_Ns7AjA 🧑⚕️📖 Stay updated with the latest in medical and health sciences—follow the Journal of Advanced Research in Medical and Health Science for more cutting-edge research. #MedicalResearch #Rheumatology #Evusheld #COVID19 #BCellDepletion #AutoimmuneDisease #HealthcareInnovation #Rituximab #ScientificBreakthrough #MedicalJournals #Research

🚨🔬The latest publication from the NECESSITY consortium sheds light on Sjögren’s disease, a condition known for its heterogeneity and lack of clear classification for the variety of symptoms observed in Sjögren’s patients. Using cluster analysis based on subjective symptoms, clinical, and biological manifestations, the study identified 3 distinct subgroups. In total 26 variables were used to cluster the population into these subgroups, to also allow for a comparative analysis of the prognosis between the three. The #findings suggest that the three subgroups represent different heterogeneous #pathophysiological disease mechanisms, stages of disease, or both. These #subgroups can be used further for the #stratification of patients in future #therapeutic trials! Read more about this groundbreaking research in our latest publication, with special thanks to Yann Nguyen, Pr Raphaèle Seror (MD, PhD), Gaetane Nocturne, PhD, Fai Ng for their efforts on this study! Our article link: https://lnkd.in/edmEgcrv #SjögrensDisease #NECESSITYConsortium #ResearchPublication #MedicalScience