FDA Decision on Sarepta's ELEVIDYS Webinar
Автор: CureDuchenneMD
Загружено: 2024-06-26
Просмотров: 2514
Описание:
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector.
This therapy, while not a cure, aims to improve the quality of life for patients. The treatment involves a single dose, but patients must undergo extensive pre-treatment screening to help mitigate the chances severe immune reactions.
Key points include understanding the genetic mutation specifics, managing potential side effects, and adhering to post-treatment protocols. The process includes genetic testing, antibody screening, and regular follow-ups. This therapy has been approved for use in the US, with ongoing studies to confirm its long-term efficacy.
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