11 March 2024
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:
Tuesday 2 April 2024, 17:00 – 18:00 CEST
Dr. Francina Munell is a Pediatric Neurologist working at Vall d’Hebrón University Hospital, Barcelona (Spain), and the coordinator of the Pediatric Neuromuscular Unit. Her main interest is the study of the early onset neuromuscular disorders. She participates in clinical trials using gene therapy in SMA and Duchenne Muscular Dystrophy, in research projects aiming to develop a therapeutic strategy for LAMA2-MD and in the elaboration of a guideline for gene therapy in neuromuscular disorders in Spain.
Laurent Servais is Professor of Paediatric Neuromuscular Diseases at the University of Oxford in the UK, and invited Professor at the University of Liège in Belgium. He graduated in medicine and pediatrics from the University of Louvain (in Louvain-la-Neuve and Brussels in Belgium) then trained as a child neurologist in the Robert Debré Hospital in Paris, France, and as a myologist at the Institute of Myology, Pitié-Salpêtrière University Hospital, Paris.
Professor Servais’ main research interests cover innovative outcome measures and clinical trials design and newborn screening (NBS). He has been involved as Principal Investigator in several clinical trials in spinal muscular atrophy (SMA), Angelman Syndrome X-linked myotubular myopathy, and Duchenne muscular dystrophy (DMD), and in leading the NBS program for SMA NBS in Belgium and in the UK. He is the coordinating investigator of two large natural history studies in Angelman in the UK and in Belgium that aims to identify and validates innovative outcome measures and biomarkers.
Watch the replay here! (hosted by ERN EURO-NMD)
Wednesday 10 April 2024, 16:00 – 17:00 CEST
Hildegard Büning is Professor of Infection Biology of the Gene Transfer and Deputy Director of the Institute of Experimental Hematology at Hannover Medical School (Germany). She served as President of the European Society of Gene and Cell Therapy (www.ESGCT.eu), is the Scientific Secretary of the German Society for Gene Therapy (www.dg-gt.de) and a Member of the Board of the American Society of Gene & Cell Therapy (https://asgct.org/). She studied Biology and obtained her PhD (Dr. rer. nat.) at the LMU Munich. Since then, she is active in the field of adeno-associated virus (AAV) vectors with a particular focus on improving the efficacy and safety of these biological nanoparticles.
Watch the replay here! (hosted by ERN EURO-NMD)
Thursday 18 April 2024, 16:00 – 17:00 CEST
Dr. Meyer studied molecular and cellular biology at the University of Berne, Switzerland followed by a post-doctoral fellowship at Nationwide Children’s Hospital, Columbus Ohio, USA developing different therapeutic approaches for the neuromuscular disease Spinal Muscular Atrophy including the now approved AAV gene therapy Zolgensma. In 2017, she established her independent laboratory at the Center for Gene Therapy at Nationwide Children’s Hospital and became Assistant Professor at The Ohio State University, Columbus, Ohio. Her research focused on the development of novel in vitro models to study disease mechanisms and generation of novel therapeutics for neurological disorders with emphasis on neurodevelopmental and autism spectrum disorders. She also optimized cerebrospinal fluid delivery methods for delivery of therapeutics including AAVs to the nervous system and translated several additional AAV gene therapies to clinic. She served and continues to serve on scientific advisory boards for patient foundations and is Chief Scientific Advisor to Alcyone Therapeutics.
Marta Kollb-Sielecka joined the European Medicines Agency in 2013. She is a Product Lead in the Therapies for neurological and psychiatric disorders office as well as a member of Advanced Therapies Medicinal Products matrix team. She holds a Master’s degree in Pharmacy from Warsaw Medical University. Prior to joining the Agency, she held a number of positions in pharmaceutical industry.
Click here to register! (hosted by ERN EURO-NMD)
Tuesday 14 May 2024, 16:00 – 17:00 CEST
Terry Pirovolakis, co-founder of CureSPG50, confronted his child’s SPG50 diagnosis in 2019. Through successful fundraising, he led ground-breaking research, treating three children within three years. Pirovolakis established Elpida Therapeutics, a corporation with a non-profit approach, focusing on gene therapies for ultra-rare conditions. Collaborating with industry leaders, Elpida targets SPG50, CMT4J, and plans to address three more diseases in 2024, utilizing profits to sustain programs. Pirovolakis extends his impact by supporting foundations, providing Gene Therapy 101 classes, and engaging in collaborations, all centered on the goal of saving as many children as possible.
Click here to register! (hosted by ERN EURO-NMD)
Thursday 23 May 2024, 16:00 – 17:00 CEST
Registration link coming soon! (hosted by ERN EpiCARE)
Wednesday 29 May 2024, 16:00 – 17:00 CEST
Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center (LUMC, the Netherlands). She played an important role in the development of antisense mediated exon skipping for Duchenne muscular dystrophy during her PhD research (2000-2004) at the LUMC. Her work currently focuses on developing antisense-mediated exon skipping as a therapy for Duchenne muscular dystrophy and rare brain diseases. This involves work in cell and animal models to improve efficiency of exon skipping, studies in muscle pathology, the identification of biomarkers, studying the basics of pre-mRNA splicing and transcript processing and the generation and detailed analysis of mouse models.
Click here to register! (hosted by ERN EURO-NMD)
Thursday 6 June 2024, 16:00 – 17:00 CEST
Registration link coming soon! (hosted by ERN EpiCare)
Thursday 13 June 2024, 16:00 – 17:00 CEST
Dr Vázquez Costa (https://orcid.org/0000-0002-3043-7938) is MD in Neurology (2012) and PhD in motor neuron diseases (2019). Since 2013 he is devoted to the care of motor neuron disease patients and in 2020 he was granted a Juan Rodés excellence contract from the Carlos III Health Institute, to coordinate the motor neuron disease unit at Hospital la Fe. He also co-chairs the ALS Genetic Spanish consortium (ALSGESCO) and is member of the Spanish research network for rare diseases (CIBERER). This unit is a referral Spanish and European centre for the treatment and research in ALS and SMA, and participates in the European Network to Cure ALS (ENCALS) and the TRICALS consortium. Dr Vázquez Costa has participated in more than 20 research projects and in more than 30 clinical trials in both ALS and SMA, and has published more than 80 articles in indexed journals in neuromuscular diseases. He has carried out research stays at the Psychiatry department of the Universitätsklinikum in Freiburg (Germany) and at the Martinos Center for Biomedical Imaging in Boston (USA) and has collaborated and collaborates with researchers from numerous research institutes and universities. Since November 2019 he is an associate professor of neurology at the University of Valencia.
Click here to register! (hosted by ERN EURO-NMD)
Thursday 20 June 2024, 16:00 – 17:00 CEST
Registration link coming soon! (hosted by ERN EpiCare)
Thursday 27 June 2024, 16:00 – 17:00 CEST
Jan Kirschner (Professor of Pediatric Neurology) has been joint coordinator of the German network for neuromuscular disorders MD-NET (www.md-net.org) since 2008. He has been involved in planning and conducting several multicentre clinical trials. He was responsible for the coordination of a large multi-centre, placebo-controlled MD-NET trial in Duchenne muscular dystrophy. Jan has established the Care and Trial Site Registry (CTSR) containing feasibility information about neuromuscular centres worldwide. Jan has coordinated the CARE-NMD project funded by the European Union (2010-2013) with the aim to improve standard of care for Duchenne muscular dystrophy throughout Europe (www.care-nmd.eu). He has a long-standing interest in the diagnosis and treatment of myopathies, muscular dystrophies, and motoneuron diseases in children and adolescence. He is an experienced clinical researcher in this area. Jan and his team have established the SMArtCARE network to collect real-world data for patients with SMA in German speaking countries (www.smartcare.de).
Click here to register! (hosted by ERN EURO-NMD)
Tuesday 2 July 2024, 15:00-16:00 CEST
Registration link coming soon! (hosted by ERN-RND)
Tuesday 23 July 2024, 15:00-16:00 CEST
Registration link coming soon! (hosted by ERN-RND)
