1. STARS-Sarizotan (Newron Pharmaceuticals, active/not recruiting)
Focused on improving Rett-related breathing episodes
2. Low Dose Ketamine (RSRT/Vanderbilt, active/recruiting)
Focused on relief of Rett-related breathing anomalies and other symptoms
https://clinicaltrials.gov/ct2/show/NCT03633058
3. Triheptanoin UX007 (RSRT/Ultragenyx/Center for Rare Neurological Diseases, active/recruiting)
12-person trial focused on chemical reactions that produce mitochondrial energy
https://reverserett.org/research/project/triheptanoin/
4. Anavex 2-73 (RettSyndrome.org/Anavex Life Sciences, active/recruiting)
Focused on improvement of neuronal function
http://www.rettsyndrometrial.com/
5. Safety of Cannabidiol Solution in Rett Syndrome (RettSyndrome.org/GW Research Ltd., active/recruiting)
Focused on evaluating the safety and efficacy of GWP42003-P in reducing symptom severity in Rett Syndrome females, aged 2 to 18 years
https://clinicaltrials.gov/ct2/show/NCT03848832
6. Trofinetide Phase 3 (RettSyndrome.org/Acadia-Neuren, anticipated)
Focused on improving neuronal signal transmission pathways from/to the brain
7. AVXS-201 Gene Therapy Phase 1 (RSRT/Novartis-AveXis, anticipated)
Cure-focused MECP2 gene therapy
https://reverserett.org/avexis-reports-rett-gene-therapy-program-avxs-201/
Keep up the good work! Much love, Kathy Hunter