
More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.
More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.

More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.

Rett Syndrome Europe welcomes the positive CHMP opinion for Daybu (trofinetide), a meaningful milestone for families affected by Rett syndrome across Europe.

Taysha Gene Therapies has shared positive longer-term interim data from its investigational gene therapy TSHA-102, with dosing now complete in the pivotal…

Neurogene has announced the successful completion of enrollment in the EMBOLDEN trial of NGN-401, an investigational gene therapy for Rett syndrome. The…