News

NGN-401, Neurogene’s gene therapy candidate for Rett syndrome, was well tolerated in the first three patients dosed in a Phase 1/2 clinical trial. The Phase 1/2 trial (NCT05898620) is enrolling girls with Rett, ages 4-10, at three sites in the U.S. The company also plans to establish…

The U.S. Food and Drug Administration (FDA) has given a regenerative medicine advanced therapy (RMAT) designation to TSHA-102, an experimental gene therapy for Rett syndrome. RMAT is given to regenerative medicines — cell therapies, therapeutic tissues, human cell and tissue products, or any combination using such therapies or products…

The Rett Syndrome Research Trust (RSRT) has partnered with Vivalink to use its Multi-Vital ECG wearable patch to monitor heart rate in people with Rett syndrome participating in the observational VIBRANT study. VIBRANT (NCT06338267) is set to enroll about 30 Rett patients at sites…

Health Canada has agreed to review Acadia Pharmaceuticals’ application seeking approval of trofinetide — sold in the U.S. as Daybue — for the treatment of Rett syndrome. The regulatory body granted the application priority review, which is reserved for submissions that address an unmet need or…

Children with the MECP2 gene-related disorders Rett syndrome and MECP2 duplication syndrome (MDS) experience atypical sensory processing, according to a recent study. Deficits in the MeCP2 protein chiefly cause Rett syndrome, whereas MDS occurs due to duplication of the chromosomal region containing the MECP2 gene. Unusual sensory effects…

The probiotic Lactobacillus plantarum PS128 was found to ease involuntary leg muscle contractions, known as dystonia, in Rett syndrome patients, according to a pilot study that tested the supplement’s impact on neurological outcomes. Noting that Rett syndrome “often involves gastrointestinal symptoms and gut microbiota imbalances,” the researchers had sought…

The Rett Syndrome Research Trust (RSRT) is teaming up with partners in industry and academia to get ready for a study called VIBRANT, which plans to test innovative ways to measure and potentially monitor the symptoms of Rett syndrome in patients, with a focus on autonomic dysfunction. The…

Unravel Biosciences has teamed up with Quality Chemical Laboratories (QCL) to manufacture materials for clinical trials of RVL001, an investigational therapy for Rett syndrome. RVL001 (vorinostat) will be tested in proof-of-concept trials in the U.S. and Colombia. Last year, the U.S. Food and Drug Administration (FDA)…

New research supports how the lack of Mecp2 protein impairs the function of healthy nerve cells in a mouse study of Rett syndrome. Specifically, the function of nerve cells was affected when the cells were grown alongside nerve-cell supporting astrocytes lacking the Mecp2 protein, mimicking the genetic defect that…

Neurogene is expanding its ongoing Phase 1/2 clinical trial testing gene therapy candidate NGN-401 in girls with Rett syndrome and making some changes to the trial’s structure to accelerate enrollment and dosing. Changes include adding more patients and removing dose staggering (one-at-a-time dosing) in the trial’s first low-dose…