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DTSTART;TZID=Europe/Helsinki:20240702T030000
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UID:4447-1719889200-1719936000@canadianrdn.ca
SUMMARY:State of the art of clinical stage gene therapies for Leukodystrophies - EURO NMD
DESCRIPTION: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 Neurology (EAN). \nDr. Caroline Sevin is a pediatric neurologist at Kremlin Bicêtre hospital. She leads the French reference center for leukodystrophies in children at Kremlin Bicêtre hospital\, France. ‪She has long-standing interests in neurodegenerative metabolic diseases\, including lysosomal\, peroxysomal and mitochondrial diseases. She has a strong background in leukodystrophies\, particularly Adrenoleukodystrophy\, Metachromatic leukodystrophy and Krabbe disease. She’s part of international consortia for MLD and ALD. CS is PI or co-PI of several clinical trials\, mostly in ALD and MLD\, using innovative therapies (gene therapy\, intrathecal enzyme replacement therapy\, small molecules). Her research activity focuses on developing in vivo/ex vivo gene therapy and small molecules approaches for leukodystrophies.
URL:https://canadianrdn.ca/event/state-of-the-art-of-clinical-stage-gene-therapies-for-leukodystrophies-euro-nmd/
CATEGORIES:Healthcare,International,Research Focused,Virtual,Webinars
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DTSTART;TZID=Europe/Amsterdam:20240723T150000
DTEND;TZID=Europe/Amsterdam:20240723T160000
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UID:4762-1721746800-1721750400@canadianrdn.ca
SUMMARY:EURO-NMD Gene Therapy Webinar Series - Genetic therapies and therapy developments for rare movement disorders
DESCRIPTION: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 clinical departments and industry. Unique patient-driven fund raising initiatives contribute not only financial input\, but also patient perspective to research programs in her group. The main topic of her research is autosomal dominant neurodegenerative diseases that have aberrant protein aggregation as a pathological hallmark. She studies molecular disease mechanisms\, identifies biomarkers and then uses this knowledge to develop novel therapies with a focus on RNA targeting antisense oligonucleotide therapies. She is the co-founder and co-lead of the Dutch Center for RNA Therapeutics that aims to develop RNA targeting therapies for patients with ultra-rare mutations.
URL:https://canadianrdn.ca/event/euro-nmd-gene-therapy-webinar-series-genetic-therapies-and-therapy-developments-for-rare-movement-disorders/
CATEGORIES:Healthcare,International,Research Focused,Virtual,Webinars
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