Vmedvil Posted January 23, 2023 Report Share Posted January 23, 2023 It seems neurogene announces FDA clearance of IND for NGN-401 gene therapy for children with Rett Sydrome, read more at https://www.businesswire.com/news/home/20230123005045/en/Neurogene-Announces-FDA-Clearance-of-IND-for-NGN-401-Gene-Therapy-for-Children-with-Rett-Syndrome Do you think that gene therapies for Rett Sydrome are going to be effective in treatment of the sydrome? Quote Link to comment Share on other sites More sharing options...
Vmedvil Posted January 23, 2023 Author Report Share Posted January 23, 2023 "A rare genetic mutation affecting brain development in girls. Despite being caused by a gene mutation, Rett syndrome is rarely inherited. Infants seem healthy during their first six months, but over time, rapidly lose coordination, speech, and use of the hands. Symptoms may then stabilize for years. There's no cure, but medications, physical and speech therapy, and nutritional support help manage symptoms, prevent complications, and improve quality of life." Quote Link to comment Share on other sites More sharing options...
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