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A lack of MECP2 protein, the chief underlying cause of Rett syndrome, prevented precursor cells from transforming into fully mature nerve cells in the early stages of brain development, a study suggests. Using 3D organoids that mimic the brain, blocking a pathway elevated by MECP2 depletion enhanced the formation…
TSHA-102, an experimental gene therapy for Rett syndrome now being tested in clinical trials, has been granted fast track status by the U.S. Food and Drug Administration (FDA). “We are pleased to receive FTD [fast track designation] from the FDA, which underscores the significant unmet medical need in patients…
Taysha Gene Therapies, which is developing TSHA-102 as a potential Rett syndrome treatment, raised $150 million in a private placement funding round, with most of the money supporting clinical testing of this gene therapy. TSHA-102 is being evaluated in the REVEAL Phase 1/2 clinical trial (NCT05606614)…
The first woman with Rett syndrome given the gene therapy TSHA-102 has experienced improvements in her sleep, breathing, and motor skills in the first weeks since the treatment. That’s according to new data announced by Taysha Gene Therapies, the company developing TSHA-102. “We believe the initial safety profile…
Parents of children with Rett syndrome are able to detect disease-specific differences in the sounds made by babies who have not yet begun to speak. That’s according to a study, “Learning about neurodiversity from parents – Auditory gestalt perception of prelinguistic vocalisations,” published in Research…
Taysha Gene Therapies has received the go-ahead to continue clinical testing of TSHA-102, its gene therapy for Rett syndrome, and now is readying to dose a second patient, the company announced. The experimental gene therapy is being evaluated in REVEAL (NCT05606614), a Phase 1/2 trial that’s recruiting up…
The International Rett Syndrome Foundation (IRSF) has launched the My Rett Ally app as a digital tool to help parents and caregivers of people with Rett syndrome, calling it “a valuable addition to the resource toolkit.” Free to those who care for Rett patients in the U.S., the…
Acadia Pharmaceuticals has expanded its licensing agreement with Neuren Pharmaceuticals to commercialize Daybue (trofinetide) — the first and only therapy approved for Rett syndrome — outside of North America. Daybue was originally developed by Neuren, which licensed its rights in North America to Acadia in 2018.
A pharmaceutical company in Italy is teaming up with scientists at the Sapienza University of Rome to find out if blocking NKCC1 — a protein that loads chloride ions into nerve cells, or neurons — may reduce brain activity in people with epilepsy and certain neurological disorders like Rett…
Long-term treatment with Anavex 2-73 (blarcamesine) provided sustained reductions in disease severity and progression for adult Rett syndrome patients, according to findings from the open-label extension (OLE) part of a Phase 2 trial. The reductions were significantly greater in patients who were initially assigned to Anavex 2-73 in…
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