RareKids-CAN: Navigating Advanced Therapies in Paediatric Rare Disease: An Introduction for Patients and Caregivers
This webinar is the first in the series organized under Key Initiative 1.3 of the RareKids-CAN strategic priority, ‘Strengthening Pediatric Rare Disease Clinical Trial Capacity and Advanced Therapy Medicinal Product […]
RareKids-CAN Advanced Therapy Medicinal Products (ATMPs) series: ATMP Webinar: A Practical Primer for Clinical Coordinators, Nurses & Pharmacists
Designed for clinical coordinators, nurses, pharmacists, and research staff working in pediatric rare disease trials, this introductory session will provide a practical overview of: 🔹 Gene therapies, cell therapies, and […]
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 […]