The World Orphan Drug Congress
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe. From cell and gene therapy, genetic testing, […]
2nd Global Cell & Gene Therapy Summit
4th International Symposium on Arthrogryposis
The International Symposium on Arthrogryposis provides a platform for learning and exchanging on the clinical management and research in Arthrogryposis Multiplex Congenita (AMC) in the areas of genetics, orthopedics, and […]
EURO-NMD Gene Therapy Webinar Series – Genetic therapies and therapy developments for rare movement disorders
Willeke van Roon-Mom is a full professor of Human Genetics, in particular of translational studies of neurodegenerative disorders. Her work is highly translational in nature, working in close collaboration with […]
State of the art of clinical stage gene therapies for Leukodystrophies – EURO NMD
Educational webinars on rare neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of […]
Gene Therapy Webinar Series: Episode 10 – Genetic therapies and therapy developments for SMA
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 […]
Gene Therapy Webinar Series – Episode 11: Safety issues in gene therapies and how to address them
Professor Francesco Muntoni is a Paediatric Neurologist with an interest in clinical, pathological and molecular aspects of neuromuscular disorders. He is the director of the Dubowitz Neuromuscular Centre, a leading […]
Gene Therapy Webinar Series: Episode 8 – Genetic therapies and therapy developments for ALS
Dr Vázquez Costa has a 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 […]
Gene Therapy Webinar Series: Episode 7 – Health technology assessment (HTA) of genetic therapies
Oriana Ciani is an Associate Professor of Practice in the Public Management and Policy, Health Economics, and HTA (Health Technology Assessment) Area at SDA Bocconi School of Management. Her collaboration […]
Gene Therapy Webinar Series: Episode 6 – Genetic therapies and therapy developments for muscular dystrophies (DMD, LGMD)
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 […]