News on the first patient dosed with their gene therapy and next steps in respect of expanding the trials…
News on the first patient dosed with their gene therapy and next steps in respect of expanding the trials…
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…