Ghent University Hospital

In the context of Autoimmune Awareness Month, the coalition of patients suffering from Myasthenia Gravis (MG) "All United for MG" urges citizens throughout Europe to unite in instigating positive change and enhancing Myasthenia Gravis management. All United for MG is officially launching a European petition to advocate for better rights for patients and their caregivers, improved access to healthcare and enhanced conditions. All United for MG was founded in 2023 to raise awareness about gMG and strengthen patients and their caregivers' rights for improved quality of life, better care and access to resources on the disease. The petition aims to bring changes in the gMG and broader Rare Disease community, calling for a renewed Rare Disease Plan under the next European Commission. BRUSSELS, March 6, 2024 /PRNewswire/ -- Following Rare Disease Day and in anticipation of the European elections, All United for MG has undertaken the initiative to mobilize society, initiating transformative changes for the patient community. Since its establishment in 2023, All United for MG has been amplifying the generalised Myasthenia Gravis (gMG) patient voice across Europe. A Call for Action, launched with European parliamentarians Tomislav Sokol and Istvan Ujhelyi in June 2023 during the first-ever Myasthenia Gravis Day, contains six actionable policy recommendations for the improvement of the visibility, recognition and treatment of Myasthenia Gravis (MG) and other rare diseases. These recommendations were supported by over 30 stakeholders, including 11 members of the European Parliament, healthcare professionals and Patient Advocacy Groups (PAGs). All United for MG is now intensifying its efforts and seeks the support of all citizens across Europe by launching a petition on change.org, to encourage the civil society to support and advocate for changes in the management of rare diseases care,especially in generalized Myasthenia Gravis (gMG). The ambition is to collect between 5.000 - 10.000 signatures in the lead-up to the second Myasthenia Gravis Awareness Day on June 2nd and the European elections. gMG is recognized as a rare chronic autoimmune disease that imposes substantial challenges on the majority of individuals affected. It causes complex and chronic physical symptoms that weaken the body's muscles and can be potentially life-threatening. Affecting facial expressions, vision, speech, chewing, swallowing, and breathing, it makes daily movements exhausting and challenging. As a rare disease, it remains difficult and time-consuming to diagnose. "Taking action at European Union level is extremely important to raise awareness and increase interconnectivity among all Member States. This enables rare disease patients and healthcare professionals from different countries to exchange data, ensuring better understanding for the disease and better social support. It is thus crucial to collectively support All United for MG initiative to underline the significant impact of MG on patients' daily lives, as well as to their loved ones. I would also like to encourage all EU citizens to sign the petition, stressing the importance of raising awareness of MG and defending the rights of those affected, and bringing positive change to the rare disease environment in Europe" said Stelios Kympouropoulos, Member of the European Parliament. "gMG is a debilitating neuromuscular disease with complex physical symptoms that, for the most severely affected patients, even necessitate the assistance of a caregiver," added Prof. dr. Jan De Bleecker, Ghent University Hospital and Ghent University. "There remain unmet needs in rare disease, and as healthcare professionals, it is crucial for us to ensure that the broader healthcare community comprehends the intricacies of the disease for improved management." "The burden of the gMG is not universally well-understood or recognised across Europe. Raising awareness and elevating the patient voice can make a meaningful difference in driving change. The aim of the petition is thus to mobilise each citizen to lend their support to patients and the broader rare disease community. By uniting individuals in this collective effort, we can foster a greater understanding, stand up for improved resources, and ultimately enhance the overall quality of life for those affected" said Lutgarde Allard, Board Member at EUMGA. "Rare diseases often struggle for visibility in political discourse, making the All United for MG petition all the more important. This Call to Action urges citizens to join hands in encouraging positive change. In the current political environment, this petition serves as a powerful tool to highlight the necessity of prioritizing Myasthenia Gravis, neuromuscular disorders and more broadly rare diseases in the policy agenda" added Jean-Philippe Plançon, President of EPODIN. If you want to express your support by adding your signature, you can access the online petition via this link: . About Generalized Myasthenia Gravis Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months, where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population. SOURCE All United for MG

Opus to advance preclinical development programs for BEST1- and RHO-related retinal diseases Deal expands Opus’ addressable patient population for its novel treatments for rare inherited retinal diseases RESEARCH TRIANGLE PARK, N.C., Dec. 28, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio. Opus will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), respectively. BEST1-related IRDs are estimated to affect approximately one in 69,000 people, or nearly 5,000 people, in the United States. The BEST1 gene therapy is designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells to produce bestrophin-1 protein and normalize homeostasis between the photoreceptors and retinal pigment epithelial cells. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone. “The addition of these innovative BEST1 and RHO-adRP programs significantly increases the patient population that could benefit from Opus therapies and complements our existing pipeline of gene therapies for inherited retinal diseases,” said Ben Yerxa, PhD, Chief Executive Officer of Opus. “Opus is building an engine for addressing multiple IRDs, and deals like this one help us achieve our desire for sustainable growth of our infrastructure, operations, scientific expertise and ultimately our patient impact, with the goal of at least one IND per year.” Opus anticipates completing additional IND-enabling studies and filing an IND for BEST1 in the second half of 2023. As part of the deal, Opus will assume responsibility for the global research, development and commercialization of BEST1 and RHO-adRP programs. In exchange, Iveric received an upfront payment of $500,000 and high single-digit percentage ownership of Opus. Iveric is also eligible to receive development and regulatory milestone payments, sales milestone payments, and a low single-digit earnout on net sales of the products. Iveric retains certain rights with respect to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP under certain circumstances. “Affecting over 28,000 people across the U.S., EU and UK, BEST1 and RHO-adRP represent a significant portion of all inherited retinal diseases and an urgent unmet need for effective treatment. IRDs are ideal targets for genetic therapies to stop the retinal degeneration and improve the lives of patients living with severe vision loss or blindness,” said Bart P Leroy, MD, PhD, head of the Department of Ophthalmology, professor of Ophthalmology and member of the Center for Medical Genetics at Ghent University and Ghent University Hospital in Belgium; and attending physician in the Division of Ophthalmology and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia. About Opus GeneticsOpus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com. Opus Media Contact:Heather Anderson6 Degrees919-827-5539handerson@6degreespr.com

UK forests could store almost double the amount of carbon than previous calculations suggest, with consequences for our understanding of carbon stocks and humanity's response to climate change, according to a new study. UK forests could store almost double the amount of carbon than previous calculations suggest, with consequences for our understanding of carbon stocks and humanity's response to climate change, according to a new study involving UCL researchers. For the study, published today in the journal Ecological Solutions and Evidence, the international team of scientists used a novel 3D scanning technique and analysis to assess the amount of aboveground biomass (AGB) -- used to derive carbon storage -- of 815 trees in a UK woodland. The team found that their results were 77% higher than previous estimates (410 t ha-1 of biomass vs 232 t ha-1). The authors say that their study could have implications for the role of forests in tackling climate change, with the potential underestimation of forest carbon stocks having both positive and negative consequences for climate policy. Study co-author Professor Mat Disney (UCL Geography and the National Centre for Earth Observation) said: "Forests currently act as a carbon sink in the UK. However, whilst our finding that the carbon storage capacity of typical UK woodland could be nearly double what we previously thought might seem like a purely positive outcome, in practice this means that for every ha of woodland lost, we're potentially losing almost twice the carbon sink capacity we thought. "This has serious implications for our understanding of the benefits of protecting trees in terms of climate mitigation -- and deforestation and afforestation targets more broadly." The study was a collaboration between researchers from UCL, UK's National Centre for Earth Observation (NCEO), the Universities of Ghent, Oxford and Tampere, The National Physical Laboratory, and Sylvera. To establish their findings, the team undertook 3D terrestrial laser scanning (TLS) analysis in a 1.4 ha section of Wytham Woods in Oxfordshire. TLS is a remote sensing technique whereby millions of laser pulses are emitted to capture the environment and structures of trees in the woodland in 3D. They then used statistical modelling to calculate the mass and volume of the trees, and subsequently the carbon storage capacity of the area, and compared this to the findings of previous models. The authors say that their study brings into question the certainty of estimates of forest carbon storage across the UK, particularly for the largest and most carbon-heavy trees, which are currently based on widely used models that estimate tree mass from the trunk diameter. It is likely that previous studies have been greatly underestimating forest biomass across the UK. Study lead author Professor Kim Calders (Ghent University) said: "Currently, most estimates of forest carbon stocks are based on simple allometric models that assume that a tree's size and mass increase at a steady rate. Our findings show that relying on these models is problematic, as they are not representative of UK forests. While the models work well for trees smaller than around 50 cm in diameter, which are fairly uniform in terms of their size and volume, this isn't what we see for larger, heavier trees. These are far more complex when it comes to structure -- and they vary hugely across place and species. "It's vital that we're able to reduce uncertainty in forest carbon estimates, given that land use, and forest protection and restoration in particular, constitute a quarter of countries' current commitments to their Paris Agreement targets." Currently, the UK's biomass stock reporting to the Food and Agriculture Organization of the UN is based on these allometric models, which the authors say have very likely resulted in significant under-reporting. Study co-author Yadvinder Malhi (Oxford University) added: "Wytham Woods belongs to the University of Oxford and has witnessed over 70 years of detailed scientific research. This research shows how new approaches can yield surprises in even well-studied forests, with profound consequences for our understanding of forests and their role in tackling climate change that apply across the UK and beyond."