Germans Trias i Pujol Research Institute (IGTP)’s Post

This time last week, Global Counsel hosted the “Rare Diseases Forum 2024: securing Europe’s competitiveness in R&D for people living with rare diseases” in Brussels to launch our latest report “Staying ahead: the case for a European action plan for rare diseases." Our key takeaways from across the panel discussions and keynotes . . . - Calls for a European action plan for rare diseases are only growing stronger, but Europe needs to take action now. Patients can't afford to wait for a plan. There was strong consensus, including from Frank Vandenbroucke, Belgian Deputy Prime Minister, that an action plan is sorely needed. - In the face of intensifying global competition, Europe should play to its strengths. Europe’s biopharma sector does not have the same level of investment as the biotech clusters in the US, but where Europe can be world-leading is in legislation and enabling fast, effective approvals. - Stop talking “about” patients, and start talking “to” them. People living with rare diseases are experts in their conditions and offer invaluable insights when patient populations are so small. Drug development, clinical research and reimbursement models need to be patient centred for the benefit of the patient and for research. Read the report at https://lnkd.in/eA8DmW3M. A huge thank you to our event partners - EURORDIS-Rare Diseases Europe, RARE DISEASES ORGANISATION BELGIUM, EFPIA - European Federation of Pharmaceutical Industries and Associations, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs, EuropaBio - the European Association for Bioindustries, pharma.be, UCB, Novo Nordisk, Sanofi Belgium and Takeda - for making Tuesday’s event a success.

How to Diagnose Mycobacteria? In the dynamic landscape of mycobacteria diagnostics, laboratories and healthcare professionals are actively exploring novel approaches to elevate precision and streamline detection. Let’s dive into some solutions for identifying both non-tuberculous mycobacteria (NTM) and the tuberculosis-causing 𝘔. 𝘵𝘶𝘣𝘦𝘳𝘤𝘶𝘭𝘰𝘴𝘪𝘴 complex (MTBC) in our new blog article. What solution do you use or what solution do you like to use? #BrukerMID #moleculardiagnostics #mycobacteria #tuberculosis #PCR 

To all Scientists working in TB, here's your opportunity to present your work at International Union Against Tuberculosis and Lung Disease (The Union) TBScience conference (in Bali!). Call for Abstracts now open https://lnkd.in/g8RRytNu The four themes are 1. Implications of pathogen heterogeneity for intervention 2. Fundamental advances in understanding pathogenesis 3. Mechanisms underlying heterogeneous disease manifestations 4. Pharmacological considerations for optimising new regimens Deadline for submission 10th June.

On this #RareDiseaseDay , let's come together to shine a light on the challenges faced by those living with rare diseases worldwide. With over 7,000 known rare diseases affecting millions, it's crucial that we stand united in our advocacy and understanding.🌟💙 Today, we applaud innovators like Moderna, dedicated to developing treatments for rare metabolic disorders like methylmalonic acidemia (MMA), propionic acidemia (PA), and glycogen storage disease type 1a (GSD1a). Their efforts offer hope and transformative solutions for those affected, inspiring the entire rare disease community. Learn more here: https://lnkd.in/gMVyvjur Let's continue to raise awareness, extend our support, and stand in solidarity with those facing rare diseases. Together, we can make a difference. 💪 #rarediseaseday2024 #Awareness #Support #Solidarity #raredisease #rarediseaseawareness #rarediseaseresearch #sciencecommunication #biotechnologie #biotech

🔬🌟 Today marks the rarest day of the year, making it the perfect occasion to raise awareness for Rare Disease Day! There are over 6000 identified rare diseases, and while each disease may affect only a small portion of the population individually, collectively they impact 1 in 17 people worldwide. Unfortunately, many rare diseases often go undiagnosed or have little to no treatment. Therefore, we felt compelled to highlight and celebrate the remarkable efforts of just a few (of the very many) healthcare and life science companies making a difference: 🔍 OGT’s solutions are at the forefront of rare disease research, profiling a range of mutations crucial to solving the puzzle of rare diseases. The company’s NGS, FISH and microarray products are designed for robust identification of the whole range of genomic variation and aim to enable researchers and clinical decision makers to reach the right decisions every time. 👶 Revvity has demonstrated huge commitment to early detection and intervention of rare diseases, with over 800 million babies screened globally using their systems. By facilitating early identification, Revvity's systems play a crucial role in enabling healthcare providers to implement timely interventions, and improve the outcomes and quality of life for countless individuals. 💊 Mereo BioPharma is a clinical-stage biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. With a portfolio covering potential therapies for conditions like Osteogenesis Imperfecta and Alpha-1 Antitrypsin Deficiency-associated lung disease, they are addressing critical needs in these underserved patient populations. From clinical research to screening and therapeutics, these are just some of the many great companies playing a crucial part in advancing our understanding of rare disease, and ultimately enhancing the quality of life for individuals affected by rare diseases. Working together, we can all make a difference!🌟 #RareDiseaseDay #ShareYourColours #Research #Healthcare #RareDiseaseAwareness

Project update 📣 We are pleased to announce the successful inclusion of our first participant in the major EU project "Respiri TB". In this ascending dose, phase 1 trial, we are evaluating the pharmacokinetics, safety, and tolerability of a single intramuscular dose of the long-acting injectable formulation of the antitubercular drug Bedaquiline in healthy volunteers. We will soon proceed with our second participant. Safety and tolerability data will be reviewed after one week before administering the drug to the remaining participants in this first dose group. Overall, pharmacokinetic assessments and safety evaluations will be conducted for up to 52 weeks post-administration. Marlene Prager, coordinating junior investigator, highlights the importance of our research: “What I find particularly exciting about this project is its potential impact on tuberculosis, a disease that continues to affect over 10 million people annually. Preventing the progression from latent TB infection to active disease through effective preventive therapy is important for at-risk populations.” She further explains that current tuberculosis preventive therapies face challenges like poor adherence due to relatively long treatment regimens that mostly require daily pills. “To address this, we are evaluating a long-acting formulation of Bedaquiline for single intramuscular injection. If this therapy proves to be safe and effective in the long term, it could make a notable contribution to the WHO's End TB Strategy. It is really fascinating to be part of such an important milestone that might effect the life of so many people…” With Markus Zeitlinger #clinicalpharmacology #tuberculosis #research #medicalresearch #EUproject

Molecular point-of-care (POC) tests could revolutionize infectious disease diagnosis in resource-limited settings. Ryan Relich and colleagues at Indiana University discuss the role of molecular POC tests in a recent review published in the Journal of Clinical Virology. They highlight the high sensitivity, rapid turnaround times, relative ease of use, and convenience of laboratory-grade testing that make them an appealing option. The authors also address the associated logistical challenges, including U.S. Food and Drug Administration approval, Clinical Laboratory Improvement Amendments complexity levels, and the REASSURED criteria. POC tests currently in research and development phases are also presented, with potential for commercialization and implementation in limited-resource settings. The authors review published studies that have assessed the clinical impact of molecular POC testing in limited- and moderate-resource settings. Check out the full article to learn more about how molecular POC tests can transform infectious disease diagnosis in resource-limited settings.

https://lnkd.in/ejY3_sy5                                                                                Article title: A Belgian Randomized Trial in Hepatitis C Virus-Infected Patients with Persistently Normal Alanine Aminotransferase   Author(s): Okyay Elkilic*, Philippe Langlet, Michael Adler, Chantal De Galocsy, Isabelle Colle, An Reekmans, JeanPierre Mulkay, Hans Van Vlierberghe, Nathalie Boon, Geert Robaeys and Luc Lasser Journal: Global Journal of Clinical Virology   Journal ISSN: 2692-4749 Abstract: Persistently normal alanine aminotransferase (PNALT) is present in 30 to 40% of chronic hepatitis patients and it is generally accepted that they have no liver damage. However, some studies suggest that there are some degrees of mild to moderate histological liver damage. We evaluated the fibrosis stage and the virological outcome in treated patients with PNALT. #HepatitisC #Treatment #NormalTransaminase #EvolutionOfTransaminasesDuringTreatment #RespiratoryInfections #ViralCentralNervousSystemInfections #GastrointestinalSyndromes #ViralHepatitis #InfectionsInOrganTransplantRecipients #ViralMyocarditis #ViralInfectionsOfTheSkin #ViralHemorrhagicFevers #ViralDiseaseOfTheEye #Peertechz #PeertechzPublications #AntiretroviralAgents #AntiherpesVirus #AntiHepatitisVirus #AntiRespiratoryVirusAgents #DiagnosisOfViralInfections #ImmuneResponsesToViruses #ImmunizationAgainstViralDiseases #GeneTherapyAndViruses #ChronicFatigueSyndrome #PostInfectiveFatigueSyndromes #SimplexViruses #CercopithecineHerpesvirus #HumanHerpesvirus #EpsteinBarrVirus #SarcomaAssociatedHerpesvirus #Adenoviruses #Polyomaviruses #HumanParvoviruses #HumanImmunodeficiencyVirus #TickFeverVirus #RespiratorySyncytialVirus #HumanMetapneumovirus #ParainfluenzaViruses #ZoonoticParamyxoviruses #Filoviruses

We are pleased to announce the publication of new preclinical data evaluating Neumifil in a wide range of viral disease models. These preclinical data are published in the peer-reviewed journal, Antiviral Research in a paper entitled, “HEX17(Neumifil): An intranasal respiratory biotherapeutic with broad-acting antiviral activity,” and confirm the potential of Neumifil as a first in class protein-based antiviral prophylactic for respiratory viral infections, which has now successfully completed a Phase 2 proof of concept influenza human challenge study. The full publication can be accessed online at https://lnkd.in/ejwdhDMj. Visit https://www.pneumagen.com to learn more about Pneumagen and its broad-spectrum antiviral candidate. #broad spectrum antiviral #infectious diseases #biotech #innovation

As the world continues to face public health challenges, Emmes remains at the forefront of vaccine and infectious disease research. With over 40 years of experience across 600 clinical trials globally, we’ve been a crucial partner in developing vaccines for pandemics and epidemics. Our extensive global footprint, robust site networks, and deep regulatory expertise enable us to conduct trials that are both rigorous and globally relevant. By integrating advanced technology and AI, we ensure the highest quality standards and efficiency in our mission to advance global health. Read more: https://lnkd.in/e5wYXerX   #PublicHealth #VaccineResearch #CRO #InfectiousDiseases #Innovation #VeridixAI #VaxTRIALS Archana Sarda, Subhrajit Ghose, Karunakar Reddy, Pankaj Dua, Dr Arpit Agarwal, Narasimha Rao Ayyalasomayajula, Rajaram Venkatesan, Sagar Sutar, Sandesh Nalawade Ph.D., MBA, PMP®, VaxTRIALS, part of Emmes Group, Veridix AI, part of Emmes Group