Rett Syndrome
Pipeline by Development Stage
Drug Modality Breakdown
On Market (2)
Approved therapies currently available
Competitive Landscape
5 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (11)
Total enrollment: 505 patients across 11 trials
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Open-Label Extension Study of Trofinetide for Rett Syndrome
A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)