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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…
For caregivers of people with Rett syndrome, symptoms related to communication, mobility, and functional hand movements are the most important targets of treatment, according to a recent survey. When asked about all the symptoms included on the Rett Syndrome Behavior Questionnaire (RSBQ) — a standard way of evaluating Rett…
More than a quarter of men at large may carry mutations causing Rett syndrome in a portion of their sperm, a study suggests. The study, “MECP2 germline mosaicism plays an important part in the inheritance of Rett syndrome: a study of MECP2 germline mosaicism in males,”…
Anavex Life Sciences has completed dosing all 92 girls with Rett syndrome taking part in a clinical trial testing the safety of its oral treatment candidate Anavex 2-73 (blarcamesine). Top-line data from this Phase 2/3 study of Anavex 2-73 — which aims to ease the symptoms of the…
The first patient has been dosed with TSHA-102, an investigational gene therapy by Taysha Gene Therapies, to treat Rett syndrome. The Phase 1/2 trial, dubbed REVEAL (NCT05606614), is still recruiting up to 12 women with Rett syndrome at its single site in Montreal, Canada. Early safety data…
Levels of anxiety and depression in girls and women with Rett syndrome are influenced both by the individual’s type of MeCP2 gene mutation — the underlying cause of the neurodevelopmental disorder — and the presence of sleep problems, a new study reports. In particular, patients carrying p.Arg294*, a mutation…
TSHA-102, an experimental gene therapy by Taysha Gene Therapies, can increase the levels of MeCP2 — a protein that’s not found in sufficient amounts or is dysfunctional in people with Rett syndrome — while causing no harm to cells that have normal levels of the protein. That’s according…
The Phase 1/2 REVEAL study, which is testing the investigational gene therapy TSHA-102 in women with Rett syndrome, is expected to dose its first participant in the next month or two, according to Taysha Gene Therapies, its developer. “Screening is completed, and dosing is now scheduled for our first…
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