Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome

[Vmedvil]

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?

[Vmedvil]

"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."