Rett Syndrome
Kezar Initiates Strategic Review After FDA Setback; Taysha Regains Full Rights to Rett Syndrome Program as Astellas Agreement Ends
Kezar Life Sciences; FDA setback; strategic alternatives; zetomipzomib; autoimmune hepatitis; Taysha Gene Therapies; Astellas; Rett syndrome; TSHA-102; clinical trials
Neurogene Details New Safety Measures After Young Patient’s Death in Rett Syndrome AAV Gene Therapy Trial
Neurogene; AAV gene therapy; Rett syndrome; NGN-401; ASGCT; safety measures; HLH; clinical trial; MECP2 gene; FDA; dose adjustment
Neurogene’s Rett Syndrome Trial Halted After Patient Death Linked to High-Dose Gene Therapy
Neurogene, Rett syndrome, gene therapy, AAV, clinical trial, patient death, high-dose complications
Neurogene’s Rett Syndrome Gene Therapy Shows Efficacy but Raises Safety Concerns
Neurogene, Rett syndrome, gene therapy, NGN-401, AAV vector, clinical trial, safety concerns, efficacy results
Taysha Gene Therapies Announces Positive Results for TSHA-102 in Rett Syndrome Trials
Taysha Gene Therapies, TSHA-102, Rett Syndrome, Gene Therapy, Motor Skills, Communication, Autonomic Function, Seizures