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EAN Virtual Congress -Symposium 2: EAN/MDS-ES: Movement disorders in the emergency room
Prof. Teresinha Evangelista spoke about genetic therapy in neuromuscular disorders. She described the new genetic therapeutic approaches to the treatment of peripheral neuropathies, hereditary transthyretin amyloidosis and limb-girdle muscular dystrophy. She concluded by highlighting that gene therapies are likely irreversible, potentially providing sustained benefits but also carrying a possible risk of long-term adverse effects.