Labiotech.eu’s Post

Our goal is to make therapies accessible for the >90% of people with a #raredisease who lack them. Join us on October 1st for the 1st Annual Respiratory Rare Disease Accelerator Day at The Francis Crick Institute. 👉 https://lnkd.in/ezN7RDuz Globally, more than 300 million people are living with #rarediseases, of which one in 20 are related to #respiratory conditions. While rare on their own, collectively they represent significant social and economic costs. Given more than 90% of rare diseases lack an FDA-approved treatment, rare respiratory diseases represent a phenomenal growth opportunity. With our new LifeArc Centre for Rare Respiratory Diseases, we embody this bold ambition to improve diagnosis and care for patients living with rare respiratory disease and foster innovation for much-needed treatments and cures. Leading with three disease exemplars, we aim to de-risk translation from discovery through to clinical trials. We showcase primary ciliary dyskinesia (PCD), which affects 1:7000 births, which impairs mucociliary clearance leading to decline in lung function similar to #cysticfibrosis (CF). Unlike #CF, no approved treatments exist for PCD, despite a similar market size and unmet significant need. Together, we aim to change this.   This event will have wider applicability across rare disease and #personalisedmedicine showing how we can connect all the dots to market. With support from Weatherden, LifeArc, AXON and Pinsent Masons #Raredisease #CysticFibrosys #PCDResearch #genetherapy #respiratorymedicine #translationalmedicine #CRISPR #genomics #MolecularDiagnosis #bronchiectastis

Are you attending the European Congress on Obesity in May? Don't miss the chance to meet #Perspectum's Pharma Solutions team! Book and appointment with one of our team members to learn about our leadership in using imaging to identify and monitor multi-organ disease in clincal trials. Let us help you revolutionize your approach to clinical trials in obesity-related liver diseases. See you there! #ECO24 #clinicaltrials

Researchers have discovered a new drug that shows promising results in slowing down the progression of Chronic Kidney Disease. A groundbreaking clinical trial led by Dr. Katherine Tuttle, executive director for research at Providence Inland Northwest Health, showcases significant advancements in kidney disease treatment. This is incredible news for those who struggle with this condition. Learn more about the study's findings and the potential of the new drug, BI 690517: http://ms.spr.ly/6040iiFuM #healthcareresearch #healthcareleaders

LIVERFASt provides all the 3 critical components of NASH especially an equivalent to ballooning which can be priceless in initial diagnosis to make treatment decisions and for serial monitoring (the latter needing some validation) as pharmacotherapy takes on the new chapter of this science. The burden of cost of missing advanced liver fibrosis in a disease which is endemic to almost all populations is tremendous The burden of false positives when combining NITs is also tremendous as it can lead to more unnecessary liver biopsies which are invasive and associated with significant complication rate. Combination of LIVERFASt and LSM outperforms Fib4 and LSM across the spectrum of liver fibrosis. LIVERFASt can serve as both a screening and a diagnostic test in patients where there is a high prevalence of the disease and potentially minimize the false negative and false positive rates thereby reducing cost and helping diagnose disease accurately. #liverfast #noninvasivetesting 

Recently, the FDA unveiled the Rare Disease Innovation Hub – a new model that aims to foster collaboration and advance outcomes for patients with rare diseases. We're grateful for the FDA’s commitment to this area and with our like-minded vision to improve the patient journey, we recognize the critical need to break down barriers and co-create solutions to increase equitable care for all patients – no matter who they are or where they live. Working to enhance patient outcomes is at the core of what we do and why we are committed to activating meaningful change and empowering innovation across the rare disease care paradigm. The establishment of the Innovation Hub signifies a step forward toward optimizing care for patients, and ultimately creating a brighter future for the rare disease community. Read the FDA’s full statement: https://lnkd.in/eT3ZEEge

Today, on World Sickle Cell Day, we stand in solidarity with individuals and families affected by sickle cell disease, reaffirming our commitment to advancing diagnostics and improving patient care. At Diazyme Laboratories, we understand the critical importance of early detection and monitoring in managing this complex condition. Studies show the crucial role Ferritin Assays play in the diagnosis and management of sickle cell disease by providing accurate and reliable measurements of ferritin levels. Elevated Ferritin levels are often indicative of increased iron levels in the body, a common complication in sickle cell disease that can lead to serious health issues. On this meaningful day, we recognize the resilience of those living with sickle cell disease and the tireless efforts of healthcare professionals dedicated to their care. Through our innovative diagnostic solutions, we strive to empower healthcare providers with the tools they need to improve patient outcomes and enhance quality of life. Together, let's raise awareness, foster compassion, and advocate for better access to care for individuals and communities affected by sickle cell disease. Contact a rep to learn more: https://bit.ly/2BuMrVS **Diazyme’s Ferritin Assay is FDA approved he quantitative determination of ferritin in human serum, K2EDTA plasma, and lithium heparin plasma on Hitachi 917 analyzer. For in vitro diagnostic use only. ** #WorldSickleCellDay #SickleCellAwareness #DiazymeLaboratories #FerritinAssay #Healthcare #Diagnostics #Innovation #PatientCare #CommunitySupport

A big thank you to all of you who listened in on our virtual KOL event yesterday and asked your questions! 🙏🏼 Thank you also to Karaa, Amel,M.D. and Regina, one of her patients, who joined us to talk about primary mitochondrial disease and how it impacts the daily lives of those affected. The unmet medical need is huge, and we hope to be one step closer to finding a treatment. 💊 Watch the full event replay by clicking the link below: #mitochondrialdisease #mito #drugdevelopment #clinicaldevelopment #raredisease #patientsfirst #imp #umdf #mitocon https://lnkd.in/dpmbxWih

ALPCO’s new infographic highlights the American Gastroenterological Association (AGA)’s clinical practice guidelines, particularly focusing on the use of a biomarker and symptom-based monitoring strategy. It clarifies the significance of fecal calprotectin and C-reactive protein (CRP) levels in assessing disease activity and guiding the need for endoscopic evaluation.      Learn more here: bit.ly/3V7Xmvx #AGA #AGAGuidelines #Biomarkers #CrohnsDisease #CrohnsResearch #CrohnsAwareness #CrohnsManagement #CD #CDResearch #CDAwareness #CDManagement

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

𝗔𝘁 𝗮𝗻𝘆 𝗴𝗶𝘃𝗲𝗻 𝘁𝗶𝗺𝗲, 𝟰% 𝗼𝗳 𝘁𝗵𝗲 𝗴𝗹𝗼𝗯𝗮𝗹 𝗽𝗼𝗽𝘂𝗹𝗮𝘁𝗶𝗼𝗻 𝗴𝗿𝗮𝗽𝗽𝗹𝗲𝘀 𝘄𝗶𝘁𝗵 𝗮 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲. Yet, of the estimated 10,000 rare diseases that have been identified, a staggering 95% lack FDA-approved treatments, leaving a vast majority searching for cures. These numbers underscore the intricate web of challenges in drug development for rare diseases—from diagnosis hurdles to regulatory pathways and the quest for sustainable treatments. Today, at the 19th Annual HD Therapeutics Conference, hosted by the CHDI Foundation, Alessandro Piai highlighted IRBM’s integrated approach and how we leverage our experience to drive even more progress toward the discovery of new therapeutics for rare diseases. 🧬 On this @Rare Disease Day, let's all recognize those who are affected by rare diseases and recommit to transforming the landscape of drug development, treatment, and care. Michele Luche Sara Tomaselli Carlo Toniatti Manuel Daldin Cristina Cariulo #rarediseaseday #huntingtonsdisease #irbm #hopeforacure