To mark Rare Disease Day 2025 we are sharing the amazing adventures of Selena – an very inspiring young lady with Rett syndrome and her equally wonderful family!
To mark Rare Disease Day 2025 we are sharing the amazing adventures of Selena – an very inspiring young lady with Rett syndrome and her equally wonderful family!
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…