News

Rett gene therapy NGN-401 selected for FDA’s START program

The U.S. Food and Drug Administration (FDA) has selected Neurogene‘s NGN-401, an experimental gene therapy for Rett Syndrome, for its Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. As part of START, which the FDA launched in September 2023, Neurogene will have enhanced communications with…

MeCP2 regulates production of protein tied to Parkinson’s: Study

MeCP2, the protein impaired in most cases of Rett syndrome, controls the production of alpha-synuclein, a protein implicated in Parkinson’s disease, a study reports. Alpha-synuclein production was highest when the MeCP2 protein carried mutations from Rett patients with symptoms similar to those of Parkinson’s, data showed. “MeCP2 is…

FDA moves to support TSHA-102, potential Rett gene therapy

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…

PS128 probiotic found to ease leg muscle contractions in Rett patients

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…