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We are delighted to announce that the 8th Annual Meeting of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD) will take place from March 5 to 7, 2025 in Essen, Germany.
This event remains an essential platform for advancing research, sharing knowledge and promoting collaboration within the neuromuscular disease community.
More info to come, so stay tuned!
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Dear ERN community, In an effort to support the transition from the current CPMS to the new one, we have produced some videos, two so far, which you are free to distribute to anyone who might be interested. The first one is a more general overview of the system, how it works and what you can do inside it. This video can be found here: https://webgate.ec.europa.eu/ern/imgs/CPMS2.mp4
The second video goes through the process of signing up both as an ERN Clinician, or a guest.
https://webgate.ec.europa.eu/ern/imgs/signup.mp4
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We are delighted to share the inspiring journeys of courageous people living with neuromuscular disease. Their stories are a testament to the resilience, strength and power of community support.
A innovative collaboration between patients and clinicians aimed at producing insightful Patient Journeys focused on three pathologies: Myasthenia Gravis, Myofibrillar Myopathy, and Multifocal Motor Neuropathy. This innovative initiative seeks to shed light on the unique experiences, challenges, and triumphs of individuals living with these conditions. Each Patient Journey will offer a unique perspective, delving into the diagnosis process, treatment strategies, daily challenges, milestones, and moments of resilience and hope. These narratives will serve as invaluable resources for patients seeking support, clinicians striving for greater insight, and researchers pursuing innovative solutions.
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We would like to inform you that we will be performing important maintenance on your server this summer. Details of the intervention: - Date: Tuesday, July 30
- Time: From 8 AM to 6 PM (the duration of the intervention could be shorter)
- Impact: Your platform will be unavailable during this period.
Purpose of…
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Neuropathies is bringing together professionals from various specialties to shed light on the complexities of neuropathic disorders. It's a vibrant platform where neurologists, geneticists, pathologists, and other experts gather to illuminate the latest research findings and clinical implications. Through lively discussions and collaborative exchanges, attendees ignite new ideas and approaches to enhance patient care.
This gathering isn't just informative; it's a ray of hope for individuals affected by neuropathies, promising brighter prospects through shared knowledge and teamwork. This year, Tallinn, the enchanting capital of Estonia, with its rich history and vibrant culture, provides the perfect backdrop for the gathering, that is aimed to rotate between Baltic countries every year in August.
📆 23 - 24 August 2024
📍 Tallinn, Estonia
Click here for the Programme!
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On behalf of Mitocon and its Scientific Program Committee, we are pleased to announce the organization of the 14th Mitochondrial Disease Conference, which will be held on October 25-27, 2024 at the Best Western Hotel Galileo Padova.
As usual, the event will consist of two parts: the scientific meeting, 25-26 October and the patient and family meeting on Sunday 27 October. During the Scientific Conference, the participation of patients and families will also be encouraged. The Mito Conference 2024 aims to provide a comprehensive overview of the latest advances, challenges, and potential opportunities in the field of mitochondrial research. By covering diverse topics ranging from basic science to mitochondrial biology, with a focus on therapies and the role of artificial intelligence in mitochondrial medicine. Together, let’s light the way to a cure for all mitochondrial diseases!
Click here for the Programme!
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We’re happy to announce our upcoming webinar series on Gene Therapy co-organized by EURO-NMD, ERN-RND, for rare neurological diseases, ERN EpiCARE, for rare and complex epilepsies and European Academy of Neurology (EAN), where we’ll delve into the latest advancements and breakthroughs in this interesting field. Join us as we will explore: - Cutting-edge research in gene therapy: Discover the latest developments and innovations driving gene therapy forward.
- Clinical applications and successes: Learn about successful gene therapy treatments and their impact on patients’ lives.
- Ethical considerations and future prospects: Delve into the ethical implications of gene therapy and explore future directions for research and application.
- Expert discussions and Q&A sessions: Engage with leading experts in the field, participate in insightful discussions, and get your questions answered.
Tuesday 2 July 2024, 15:00 – 16:00 CEST "State of the art of clinical stage gene therapies for leukodystrophies" by Dr. Caroline Sevin (Kremlin Bicêtre Hospital, France).
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You can also register for the last episode (ep. 12) now:
Tuesday 23 July 2024, 15:00 – 16:00 CEST Episode 12: "Genetic therapies and therapy developments for rare movement disorders (HD, SCA)" by Dr. Willeke van Roon-Mom (Leiden University Medical Center, The Netherlands) Click here to register!
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Click here fore more information on the other episode of the series and to see the full schedule!
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If you missed a webinar, don't panic! All our webinars are recorded and made available on our website. |
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The 10th annual congress of the European Academy of Neurology will take place in Helsinki, the capital of Finland, on 29 June – 2 July, 2024. a great opportunity for networking, learning, and sharing expertise. If you're planning to attend, be sure to register early and make your travel arrangements in advance to make the most of this prestigious event.
We will have a booth to represent EURO-NMD, It's a valuable opportunity to engage with attendees, share information about EURO-NMD's activities, and foster collaborations and partnerships with other professionals and organizations in the neurology community, more information to come!
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Developing Therapies in Rare Neuromuscular Disease: A symposium by the Advisory Committee for Therapeutics 15th anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT) Their first symposium by the Advisory Committee for Therapeutics will be a day filled with insightful talks, reflecting on the wealth of knowledge gleaned over the past 15 years of TACT’s operation. The symposium is designed for industry professionals involved in developing treatments for rare neuromuscular diseases and offers a unique opportunity to learn from past mistakes, engage with leading experts, and gain valuable insights into the challenges and opportunities within the field. Attendees can expect interactive sessions and opportunities to pose questions to the speakers, fostering meaningful dialogue and knowledge exchange. Tickets are now available! Don’t miss this unparalleled opportunity to celebrate 15 years of TACT and chart the course for future advancements in rare neuromuscular disease therapeutics.
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The World Muscle Society is a dynamic community that aims to promote, disseminate, and share all aspects of neuromuscular physiology and diseases, from basic science to patient care. It encompasses a broad range of scientists and healthcare professionals who share the common interest for neuromuscular disorders. The 29th Annual World Muscle Society Congress will be held in the beautiful city of Prague from October 8 to 12, 2024. The congress will once again be a hybrid event, offering the flexibility of virtual participation, but please feel free to attend in person to maximize the benefits of networking and scientific exchange. For this congress, they have chosen a theme centered on acquired myopathies. They hope this theme will broaden the traditional focus on inherited muscle diseases and bring new experts into our field of myopathies.
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Abstract
Spinal muscular atrophy (SMA) is one of the most common genetic diseases and was, until recently, a leading genetic cause of infant mortality. Three disease-modifying treatments have dramatically changed the disease trajectories and outcome for severely affected infants (SMA type 1), especially when initiated in the presymptomatic phase. One of these treatments is the adeno-associated viral vector 9 (AAV9) based gene therapy onasemnogene abeparvovec (Zolgensma®), which is delivered systemically and has been approved by the European Medicine Agency for SMA patients with up to three copies of the SMN2 gene or with the clinical presentation of SMA type 1. While this broad indication provides flexibility in patient selection, it also raises concerns about the risk-benefit ratio for patients with limited or no evidence supporting treatment.
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In biomedical research, particularly for rare diseases (RDs), there is a critical need for model organisms to unravel the mechanistic basis of diseases, perform biomarker studies and develop potential therapeutic interventions. Within Solve-RD, an EU-funded research project with the aim of solving large numbers of previously unsolved RDs, the European Rare Disease Models & Mechanisms Network (RDMM-Europe) has been established.
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ERDERA is excited to announce two career opportunities for experienced professionals passionate about making a difference in rare disease research. They are looking for 2 Project Managers to join their dynamic team, starting from 01/09/2024 at Biopark, Paris (with partial remote work): - Project Manager - Funding & Education: Oversee collaborative research funding and training initiatives within the ERDERA programme. Your role will involve coordinating activities, supporting research teams, and ensuring the successful execution of funding and training programmes.
- Project Manager - Data & Knowledge Hub: Oversee data management and expertise services. Your responsibilities will include coordination and support of activities on rare diseases data infrastructures, as well as assistance to the methodological and regulatory expertise hub.
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