Tuesday, Taysha Gene Therapies Inc (NASDAQ:TSHA) released longer-term clinical data from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome.
Rett syndrome, caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.
REVEAL Phase 1/2 Adolescent and Adult Trial showed sustained and new improvements observed across multiple clinical domains relative to baseline, as of 52 weeks post-treatment for ...