News

Neurogene has received written agreement from the U.S. Food and Drug Administration (FDA) for a registrational trial of NGN-401, a gene therapy candidate for Rett syndrome. A registrational, or pivotal, trial is designed to provide the evidence needed to support an application for FDA approval. “We appreciate the…

An aspiration event— when something other than air enters the lungs — increases the risk of lower respiratory tract infection and respiratory failure in people with Rett syndrome, a study found. Patients with known or suspected aspiration events more commonly had risk factors like epilepsy and difficulty swallowing, and…

Changes in auditory event-related potentials, or AEPs, the brain’s electrical activity in response to sound, may serve as a non-invasive biomarker to assess treatment response in people with Rett syndrome, a study suggested. Depressed AEPs, as measured by electroencephalography (EEG), which detects the brain’s electrical activity, were normalized when…

Better access to dental care and resources can improve quality of life for girls with Rett syndrome, according to their parents. Parents interviewed for a study on oral health-related quality of life (OHRQOL) also said their own motivation and empowerment to address their daughters’ oral healthcare concerns affected overall…

The U.S. Food and Drug Administration (FDA) agreed to Taysha Gene Therapies’ proposed design for the second part of a clinical trial testing TSHA-102, the company’s gene therapy for Rett syndrome, that’s expected to start this year. The study design is based on results from Part A of the…

Girls with Rett syndrome have significantly smaller brain volumes across all its regions, regardless of their age, a MRI study shows. Reductions in the size of certain brain regions were tied to the clinical severity of Rett patients, particularly in gross motor function, such as sitting and walking. Still,…

Taysha Gene Therapies has reached an agreement with the U.S. Food and Drug Administration (FDA) on the design and next steps to start the pivotal Part B of its ongoing Phase 1/2 REVEAL trial program, which is evaluating TSHA-102 in children, adolescents, and adults with Rett syndrome.

Bone mineral density, an indicator of bone strength, is significantly lower in girls and young women with Rett syndrome compared with healthy girls and women of the same age, a study shows. Reduced bone mineral density was tied to small body size, impaired mobility, and the use of the…

Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome. The company has submitted an application seeking regulatory clearance…

NLX-101 improves breathing and cognitive function in a female mouse model of Rett syndrome, a study found. The potential therapy activates the receptors that bind serotonin, a signaling molecule found at low levels in the brains of Rett patients. “This work provides compelling evidence of the therapeutic potential of…