Beacon for Rare Diseases’ Post

In today's #ManuscriptMonday, Emeli Chatterjee and a team of researchers studied the role of extracellular vesicles from patients with cardiorenal syndrome on renal injury using the Kidney-Chip. https://hubs.ly/Q02K2ZkG0 To learn more about their work and how Organ-Chips can be used to generate novel insights into disease mechanisms and therapeutic targets, register for our August 28th webinar: https://hubs.ly/Q02K316l0

The first in a series of Atomic IBD posts with my thoughts after ECCO. We are starting with the PROFILE study, the clinical aspects, the failure of the biomarker and the implications for our global IBD community.

A feature of real-world data is the large amount of missing covariate information at some or all visits. This recent AAPS journal publication, combine multiple imputation with a pooled test statistic to iteratively build the covariate model to capture and predict individual disease progression in rare neurological diseases (RND) patients. Find out more on how the publication in addition to accounting for individual parameter uncertainty using conditional distributions and evaluating the effect of covariates, investigated how including population parameter uncertainty influences predictions and prediction intervals. https://lnkd.in/etnXDWsf American Association of Pharmaceutical Scientists (AAPS) | @aapscomms Niels Hendrickx France Mentre Andreas Traschütz,  Cynthia Gagnon, Rebecca Schüle, ARCA Study Group, EVIDENCE-R. N. D. consortium, Matthis Synofzik, Emmanuelle Comets #Covariate Model Building #AAPS Journal

Join us for our webinar discussing metabolomic profiling of Parkinson's disease. Our expert will detail how their study compared treated and untreated patient's #metabolomic profile and incorporated #bioinformatics to uncover mechanisms of disease. https://mtbln.co/bboc0i

Our latest review sheds light on the burgeoning role of ferroptosis in neurodegeneration, including Parkinson's disease. We've highlighted regulators of ferroptosis, evidence in disease, and current therapeutic targets and clinical trials. Our perspective on the outstanding questions in the field is a must-read for anyone interested in this area of research. #neurodegeneration #Parkinsons #ferroptosis #clinicaltrials #sanofi https://lnkd.in/eEbgxXF7

Supporting our Pharma partner's precision medicine strategies in both oncology and chronic diseases settings

March is #AutoimmuneAwarenessMonth, a campaign by The Autoimmune Association which seeks to increase understanding of autoimmune disease.   Did you know that autoimmune disease is now one of the most common disease categories, ahead of cancer and heart disease?   According to the University of Oxford, a population-based study of 22 million people published in The Lancet shows that autoimmune diseases affect about one in ten individuals in the UK.   Despite improvements in the treatment of autoimmune disease through immunosuppression, current therapies are limited in their therapeutic potential.   Sitryx is leading the way in the field of immunometabolism, an exciting area of medicine that has the potential to transform the field of immunology with the development of treatments that provide lasting disease remission in major autoimmune indications with high unmet need.   The Company is building a broad pipeline of first- and best-in-class therapies that leverage metabolism to modulate immune cell function across a range of autoimmune indications including atopic dermatitis, inflammatory bowel disease and pulmonary fibrosis.   Join us in raising awareness and communicating the urgent need to advance research for autoimmune disease. Learn more here: https://lnkd.in/eJY_g7TH   #Biotech #CellMetabolism #DrugDiscovery #DrugDevelopment #Immunometabolism #Biotechnology

We are thrilled to provide a positive update on our multi-center, open-label Phase 2a clinical study evaluating OMT-28 in Primary Mitochondrial Disease (PMD) patients. The PMD-OPTION study has begun to enroll patients in the first observational part of the trial at clinical sites in Italy and Germany. The start of the PMD-OPTION study marks a major milestone in OMEICOS’ strategy to tackle diseases associated with impaired function of the mitochondria.   Progress of our study coincides nicely with this week’s #WorldMitochondrialDiseaseWeek, a global campaign raising awareness for rare mitochondrial diseases. We are committed to bring a novel, first-in-class therapeutic strategy closer to the PMD patient community.   OMT-28 has shown the potential to target a key regulator network for cell metabolism and mitochondrial function, which could translate into benefits for PMD patients and improve their quality of life.   #MitochondrialDisease #WorldMitoWeek #mito  

For clinical trials, certain exploratory outcomes can help discover important new insights into the effectiveness of the intervention, and to design subsequent trials. An interesting example is the COPDGene study (the largest #COPD study in the US involving more than 10,000 patients), for which Thirona has been running the quantitative image analysis for the entire main study. While COPD studies are generally focused on the development of emphysema and airways remodeling, we have additionally explored pulmonary vascular markers to investigate the involvement of the pulmonary arteries and veins in the development of COPD.  Learn more about the project: https://lnkd.in/ehs-icst