LFB’s Post

Today we announced positive topline results from our pivotal phase 3 REVEAL-2 clinical trial which evaluated elegrobart in chronic thyroid eye disease (TED). These results build on the positive topline findings from our REVEAL-1 clinical trial in active TED, shared earlier this year.   These encouraging findings represent a major step forward in our efforts to address the daily challenges and unmet needs of people living with active and chronic #ThyroidEyeDisease.   Learn more in the press release: https://lnkd.in/emjRqDwd

[von Willebrand Disease |100 Years] On the occasion of World Haemophilia Day, which highlights the importance of diagnosis, we can see just how far we have come over the last 💯 years in understanding and identifying von Willebrand disease. 🔬 From the first description of the disease to today’s ability to differentiate subtypes and tailor care, progress has been significant. And yet, there is still a long way to go, as many patients remain undiagnosed. Behind this reality, there are personal journeys. 🎥 Audrey’s story reminds us of what diagnosis truly means for patients and their families, not just a medical answer, but a turning point. As Professor Erik Berntorp emphasises, the challenge now is clear: 👉 Improve awareness 👉 Promote earlier diagnosis 👉 Ensure better access to care 🤝 This is a collective effort alongside healthcare professionals, researchers, industry, and patient organisations such as the World Federation of Hemophilia / Fédération mondiale de l'hémophilie, which aims to improve recognition and support patients worldwide. Belgian hemophilia society AHVH 💪 Together, we continue Building Hope Together. #BuildingHopeTogether

April is Fabry Disease Awareness Month. Swipe to the final slide to hear from the Australian Fabry Community. Fabry disease is a rare, inherited condition that often goes unrecognised, but early diagnosis can make a meaningful difference to patient outcomes. Raising awareness is key to helping more people access the care and support they need. This month, we’re shining a light on Fabry disease because awareness leads to understanding, earlier intervention, and better lives. Let’s keep the conversation going.   #ChiesiAU #FabryDisease

For many people living with a rare disease, the path to diagnosis and care is long and complex — often described as a diagnostic odyssey. Symptoms may appear without a clear cause, leading to repeated consultations, multiple specialists and, frequently, misdiagnosis. This can delay access to appropriate care and create uncertainty, anxiety and frustration. Even after diagnosis, the journey continues. Patients often face complex care pathways, limited treatment options, and the ongoing challenge of coordinating care while managing the impact on daily life. The emotional burden can be significant, and connection with patient organisations and the wider community plays a vital role in providing support and understanding. Raising awareness of the rare disease journey is essential to improving recognition, strengthening care pathways and better supporting patients at every stage. At Neopharmed Gentili Rare Disease, we are committed to supporting the rare disease community and contributing to greater understanding of the challenges patients face. Together we stand: Connected to Rare, Committed to Life. #RareDisease #PatientJourney #DiagnosisMatters #RareDiseaseCommunity #NeopharmedGentiliRareDisease

Indeed navigating the pathway to treatment for rare diseases is rarely straightforward. It’s not just the clinical complexity—it's the emotional weight carried by patients and their carers, the prolonged and often uncertain timelines, and the frequent challenge of securing funding.

#Breaking: We are excited to share positive results from our Phase III trials evaluating our investigational BTK inhibitor in people living with relapsing multiple sclerosis (RMS). For the more than 2.9 million people living with multiple sclerosis (MS) worldwide, this data brings our research and development strides into focus, representing a potential new approach to MS with comprehensive disease control. Learn more: http://spr.ly/6046BB8uD8

What can we learn from the very first participants in the FON Legacy Data Registry? 🔍 Our latest Research Explained breaks down new findings from the first FON publication and shows how shared data can generate insights that improve care for individuals living with single ventricle heart disease. 🔗 Find the Research summary and peer-reviewed article on our website: https://lnkd.in/eFUmYyib

My favourite part of every Canadian Respiratory Conference hosted by Canadian Thoracic Society / Société canadienne de thoracologie is when I get the privilege to award the Canadian Lung Association Inspiration Award. This award is given out to the poster presentation that is newsworthy and inspiring in terms of the potential to create change. We were so excited to hear the work being done by Massimo Catena from McGill University entitled 'The Performance of a Machine Learning (ML) Algorithm for Interpretation of Spirometry in Community Practice' and excited to see the potential for this innovation to shape diagnoses of lung disease in the future. #CRC2026  

🩸 The E1910 trial is changing how clinicians think about consolidation therapy in Ph− B-cell ALL. In this expert overview, Dr. Anthony Stein of City of Hope discusses the role of blinatumomab consolidation in MRD-negative disease and the potential impact on evolving standards of care in adult ALL. Key topics include: • E1910 trial insights • MRD-guided treatment strategies • Blinatumomab in frontline ALL • Implications for long-term disease control 🎥 Watch the full video on #Vumedi: https://lnkd.in/eAJ6c_w2 #AcuteLymphoblasticLeukemia #MRD

This content is intended for US audiences only. Today, we’ve announced Phase III trial data marking a significant step towards a potential new biologic treatment option for people living with chronic obstructive pulmonary disease (COPD). High-level results from the MIRANDA trial reinforce our commitment to advancing science that can help improve the lives of those living with chronic respiratory diseases. Read more here: https://bit.ly/3OMPmPy