
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…

rettX is the pan-European, patient-led Rett syndrome registry led by Rett Syndrome Europe, presented at ECRD 2026. Learn what it is and…