News

Family support weighs heavily in doctors’ decisions on ventilation

When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support are the biggest factors in their decision, a recent study reports. Many teenagers with Rett syndrome have difficulty breathing, and clinicians for some may recommend invasive ventilation via a tracheostomy…

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…