Ambagon Therapeutics’ Post

During the 2024 𝐄𝐀𝐒 𝐚𝐧𝐝 𝐄𝐀𝐒𝐋 𝐂𝐨𝐧𝐠𝐫𝐞𝐬𝐬𝐞𝐬, our talented researchers have made notable contributions by presenting their posters Alina Saidi presented "𝐋𝐢𝐯𝐞𝐫 𝐅𝐢𝐛𝐫𝐨𝐬𝐢𝐬 𝐈𝐝𝐞𝐧𝐭𝐢𝐟𝐢𝐞𝐬 𝐎𝐛𝐞𝐬𝐞 𝐒𝐮𝐛𝐣𝐞𝐜𝐭𝐬 𝐚𝐭 𝐇𝐢𝐠𝐡𝐞𝐬𝐭 𝐑𝐢𝐬𝐤 𝐟𝐨𝐫 𝐂𝐚𝐫𝐝𝐢𝐨𝐯𝐚𝐬𝐜𝐮𝐥𝐚𝐫 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: 𝐓𝐡𝐞 𝐀𝐬𝐬𝐢𝐬𝐢 𝐒𝐭𝐮𝐝𝐲." Her research emphasizes liver fibrosis as a crucial marker for cardiovascular risk in obese individuals, providing valuable insights for early intervention and management. Willy Theel MSc presented: "𝐄𝐟𝐟𝐞𝐜𝐭 𝐨𝐟 𝐏𝐫𝐚𝐯𝐚𝐬𝐭𝐚𝐭𝐢𝐧 𝐨𝐧 𝐂𝐚𝐫𝐝𝐢𝐨𝐯𝐚𝐬𝐜𝐮𝐥𝐚𝐫 𝐃𝐢𝐬𝐞𝐚𝐬𝐞𝐬 𝐢𝐧 𝐚𝐧 𝐄𝐥𝐝𝐞𝐫𝐥𝐲 𝐏𝐨𝐩𝐮𝐥𝐚𝐭𝐢𝐨𝐧 𝐰𝐢𝐭𝐡 𝐎𝐛𝐞𝐬𝐢𝐭𝐲 𝐚𝐧𝐝 𝐋𝐢𝐯𝐞𝐫 𝐅𝐢𝐛𝐫𝐨𝐬𝐢𝐬." His work was not only highly recognized but also selected as a top-rated poster presentation, underscoring its importance. His findings explore the potential benefits of pravastatin in reducing cardiovascular risks among elderly patients with obesity and liver fibrosis. For a detailed look at the presented posters, visit the MASH Clinical website: https://lnkd.in/d_QAH32A

Great news - The collaboration with EDELRIS has produced excellent results!! USP8 regulates the ubiquitination level and fate of EGFR, and its deregulation is associated with many diseases, including cancers and Cushing’s disease. Cushing’s disease is a rare and severe disease in which pituitary tumors cause an excess of adrenocorticotropic hormone (ACTH), resulting in an increased release of cortisol and adrenal androgens. New results were presented this week by Marie-Odile Fauvarque Marras (Université Grenoble Alpes, CEA, Inserm, IRIG, BGE UA13) at the EMBO workshop on Ubiquitin and ubiquitin-like proteins in health and disease. The aim of the study was to identify new ligands of USP8 to investigate the role of the USP8::CHMP1B complex in endocytosis, oncogenesis, and ACTH secretion. In the frame of the AAPG2020 DruggingUSP8 project coordinated by Marie-Odile, Edelris used its ASMS platform to screen two truncated domains, MBP-tagged MIT and RHO domains of USP8. With our expertise, specific binders of the RHO and MIT domains, originating from Edelris's +2M unique compound collection, were identified. This constitutes the first milestone in providing tool compounds to elucidate the role of USP8 in Cushing’s disease. #CushingDisease #thiscushing #biochemistry #drugdevelopment #toolcompounds #drugdiscovery

Thank you to The Kennedy Trust for Rheumatology Research for inviting me to provide a testimony for their latest booklet, titled "Building on a breakthrough: the lasting impact of biologic therapy." Seeing this booklet published in 2024 and on #NationalPatientAdvocacyDay feels quite fitting, as I 'celebrate' my 20th anniversary with anti-TNF therapy! Here is what I had to say (check out pages 10 and 11 for other testimonies from the rheumatology community): "Access to biological anti-TNF therapy in 2004 at the age of 10 transformed my life from one controlled by a disease to one where I was in control. Having lived with juvenile idiopathic arthritis from a very young age, I struggled with active disease for several years, and was intolerant to the standard of care therapy. As a consequence, I did not adhere to treatment, and had crippling anxiety at the thought of treatment. I was struggling to mobilise, and had little to look forward to in life. Within a matters of weeks of treatment with anti-TNF therapy, life began to change. I could walk. I didn’t feel sick every weekend. I began to see what life could be like." Looking back, it's no wonder that I was destined for a career in #PatientAdvocacy and #MedicalAffairs within the pharmaceutical industry... when your very existence depends on access to treatment, your outlook irreversibly changes. It's an understanding that people who don't have chronic health conditions will never truly appreciate, and is why integrated #PatientEngagement in all aspects and phases of clinical research is critical. I will forever be indebted to the patients, researchers, and healthcare professionals whose contributions helped to transform my life... and it's the reason why I continue to do what I do... so we can improve lives for the better, collectively. #MedicalCommunications #MedComms #MedAffairs #PatientAdvocate #Advocacy #PatientAdvocacy #PatientsInvolved #PatientsIncluded #NothingAboutUsWithoutUs #IntegratedPatientEngagement

Congratulations to Drew Evans (Drew Evans) on publishing his MS research in PLEFA with co-authors Jazmine Eccles-Miller (Jazmine Eccles-Miller), Hannah Farrell (Hannah Farrell) and Ellie Anderson (Eleanor Anderson). The manuscript investigates the role of lipid metabolites (oxylipins) as signaling molecules. In this case, we examined the two major products of CYP2B6; 9-HODE and 9-HOTrE and its potential to alter hepatic energy preferences and increase steatosis (fatty liver disease) in HepG2 cells. Link to the manuscript is below. https://lnkd.in/eRYKeVbc

🚀 We are excited to announce that our latest publication delve deep into uncovering vital insights into Endoglin's role in liver sinusoidal endothelial dysfunction. Our findings shed light on how changes in Endoglin expression impact liver function in cholestasis and NASH. Join us in understanding the molecular mechanisms behind liver sinusoidal endothelial dysfunction. Read the full paper and let's continue pushing the boundaries of knowledge together! 🎉 The manuscript published in a prestigious journal of Biochimica et Biophysica Acta (BBA)- molecular basis of disease [IF= 6.233], AIS 1.213 (Q1) Samira Eissazadeh Nadia(Fatemeh) Alaei Jana Urbánková Rathouská Standa Micuda Petr Nachtigal Endoglin and soluble endoglin in liver sinusoidal endothelial dysfunction in vivo - ScienceDirect

"Prevalence of Genetic Variants and Deep Phenotyping in Patients with Thoracic Aortic Aneurysm and Dissection: A Cross-Sectional Single-Centre Cohort Study", publiziert in JCM Special Issue "Diagnosis and Management of Aortic Diseases: Drafting from Theoretical Aspects to Clinical Practice" https://lnkd.in/eZVgsvBK Ich danke allen Netzwerkpartnern, gemeinsam stehen wir für innovative Gefäßmedizin 😷

Register today to join FDA’s Rare Diseases Team, NIH’s National Center for Advancing Translational Sciences, and the Reagan-Udall Foundation for a hybrid public workshop on May 13, 2024, on the use of natural history study and registry data in rare disease drug development programs: https://lnkd.in/gmJtwJbW   The workshop will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to:  ➡ address the role and design of registries and natural history studies to inform the development of rare disease treatments,  ➡ understand the considerations for collecting registry and natural history data that are fit for regulatory purposes, and  ➡ present considerations in the use of registries and natural history studies to inform regulatory decision-making. 

Dear Colleagues, A recent publication in Medical Science Monitor addresses a significant shift in the understanding and diagnosis of fatty liver disease. The paper discusses the transition from non-alcoholic fatty liver disease (NAFLD) to metabolic dysfunction-associated fatty liver disease (MAFLD), emphasizing metabolic dysfunction's role in the disease's pathogenesis. Interestingly, a 2023 proposal for metabolic dysfunction-associated steatotic liver disease (MASLD) has not shown conceptual advantages and may perform worse than MAFLD criteria. This review provides critical insights into why MAFLD remains the preferred diagnostic framework. The complete report is now available on the Medical Science Monitor webpage. | https://lnkd.in/d54tngh5

🚨 #PaperAlert 🚨 Discover the most comprehensive review on #ferroptosis to date, crafted by the Germany-wide research consortium (DFG Priority Program SPP2306) and leading external experts! This thorough examination delves into ferroptosis, its mechanisms, research tools and methods, and its potential as a therapeutic target. The review provides both seasoned scientists and newcomers with a detailed overview and inspires further research into the molecular pathways that modulate ferroptosis. Bernhard Michalke and I are proud to be part of this impactful review. 🔗 Read the full article: https://lnkd.in/gcXjtP7h #CellDeath #DiseaseResearch #RedoxBiology #ScientificDiscovery #DFG #SPP2306

Existing treatments in mucopolysaccharidosis type 1 significantly change the course of the disease but do not fully correct the phenotype. In this new paper, Drago Bratkovic and colleagues report on the use of a heparin-like macromolecular carbohydrate derivative to try to meet this unmet need. Open-label, single-center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I Drago Bratkovic, Curtis Gravance, David Ketteridge, Ravi Krishnan, Divya Navuru, CCDM®, Michael Sheehan, Donna Skerrett MD MSc, and Michael Imperiale https://lnkd.in/eBAKcGuB