Many thanks to Future Rare Diseases for sharing this publication with us. You can read the full paper here.
Many thanks to Future Rare Diseases for sharing this publication with us. You can read the full paper here.
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…