At TREAT-NMD, our mission is to foster a collaborative, inclusive global network and organisational infrastructure. Our goal is to overcome fragmentation and provide essential support services, information, and data that drive advancements in treatment, diagnosis, and care for neuromuscular patients worldwide. In line with this mission, we are proud to support the Journal of Neuromuscular Diseases (JND), a publication that plays a pivotal role in disseminating cutting-edge research and clinical findings in our field.
About the Journal of Neuromuscular Diseases
The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics, pathogenesis, pharmacology, diagnosis, and treatment of acquired and genetic neuromuscular diseases. These include muscular dystrophy, myasthenia gravis, spinal muscular atrophy (SMA), neuropathies, myopathies, myotonias, and myositis. By publishing research reports, reviews, short communications, and letters-to-the-editor, JND provides an invaluable open forum for original research in basic science, translational research, and clinical research.
Supporting such publications is key to advancing our mission at TREAT-NMD. They enable the sharing of vital information and breakthroughs that can lead to more effective treatments and improved patient outcomes. The latest edition of the Journal of Neuromuscular Diseases is now available, and we encourage everyone in the neuromuscular community to explore its valuable content. You can access the latest edition here > https://content.iospress.com/journals/journal-of-neuromuscular-diseases/11/3.
Ground-breaking Research
The latest edition of JND features their most recent study assessing the benefits of disease-modifying gene therapy treatments for vulnerable SMA patients using data from a large patient registry. This ground-breaking study, published by IOS Press, demonstrates the effectiveness of disease-modifying treatments (DMTs) in infants with SMA. The findings add substantial support for gene therapy as a treatment modality that can deliver durable transformative effects for these vulnerable patients.
Dr. Laurent Servais, the lead investigator of the study, discusses the importance of real-world data in providing evidence-based answers to parents’ questions regarding the risks and benefits of gene therapy for their children. The results confirmed that early treatment, particularly for infants identified through newborn screening, leads to better outcomes. This evidence supports the transformative potential of early intervention for SMA patients. You can read the full study here> https://content.iospress.com/articles/journal-of-neuromuscular-diseases/jnd230122
Latest Insights
The 17th UK Neuromuscular Translational Research Conference 2024 attracted over 200 basic and clinical scientists dedicated to neuromuscular disease research. Designed to promote and share cutting-edge clinical and scientific advances, the event encouraged new research and collaborations through guest lectures with Q&A sessions, research posters, and flash presentations from early career scientists. Key topics for 2024 included the latest developments in genetic therapies, applications of exercise therapy in the era of gene therapy, mechanisms and new advanced therapeutics in autoimmune neuromuscular diseases, and new genomic advances in the field. The JND have now released the abstracts from the conference, offering insights into the latest research and developments in the field of neuromuscular diseases. You can access the conference abstracts here > https://content.iospress.com/articles/journal-of-neuromuscular-diseases/jnd249001
Advancing Neuromuscular Research
By staying informed about the latest research, we can collectively contribute to the advancement of treatment, diagnosis, and care for neuromuscular patients globally. By supporting publications like JND, we can make significant strides toward improving the lives of those affected by neuromuscular diseases.
For more information and to sign up to receive their latest updates visit: https://www.iospress.com/catalog/journals/journal-of-neuromuscular-diseases
