Up to nearly half of children and adults with Rett syndrome in the U.S. have digestive problems, resulting in added medical costs that average more than $4,400 per patient each year, a study finds. While gastrointestinal (GI) manifestations appear to represent a great burden…
A Phase 1/2 clinical trial has been cleared to infuse NGN-401, a one-time investigational gene therapy, to a third girl with Rett syndrome, its developer, Neurogene, reports. The green light given by the Data Safety Monitoring Board overseeing the clinical trial’s progress was based on data indicating…
Effective communication is a top concern for people affected by Rett syndrome or related disorders, a U.S. caregiver survey has found. Other top concerns for patients include seizures, problems with walking or balancing, loss of function in the hands, and constipation. Recent progress in understanding brain development disorders such…
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…
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…
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…
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…
Scientists may have discovered promising treatments for Rett syndrome in two small molecules, called KW-2449 and VPA, each seen to ease nerve cell deficits — thereby potentially improving the flow of information between neurons — in a lab model of the disorder. “These findings suggest that KW-2449 and VPA…
NGN-401, a new gene therapy in the pipeline from Neurogene, extended the lifespan and reduced the disease burden in mice with a form of Rett syndrome, the company announced. Researchers also observed that the therapy was safe when delivered to mice or early in the life of…
A mosaic mutation — one that is present in only some body cells — causing Rett syndrome was found in a 2-year-old boy in India, a report says. According to researchers, this is the first time this specific mutation has been reported in a male patient. The report, “…
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