NLX-101 improves breathing and cognitive function in a female mouse model of Rett syndrome, a study found. The potential…
Articles by Steve Bryson, PhD
One and a half years of daily Daybue (trofinetide) treatment safely improved communication, eye contact, and hand use among…
Computer modeling confirms the weight-based dosing strategy for Daybue (trofinetide) to treat people with Rett syndrome, ages 2…
Defects in how cells degrade old or damaged components, a cellular recycling process called autophagy, contribute to the dysfunction of…
A research team has identified Rett syndrome-specific molecules that regulate gene activity, called microRNAs, that are associated with the…
A girl with Rett syndrome participating in a clinical trial testing gene therapy NGN-401 experienced a serious, treatment-related immune…
The first four girls with Rett syndrome treated with a low dose of Neurogene‘s gene therapy candidate NGN-401…
Gene mutations that cause Rett syndrome affect the function of mitochondria, the cell’s energy producers, in human astrocytes, a…
NGN-401, Neurogene‘s experimental gene therapy for Rett syndrome, was designated a regenerative medicine advanced therapy (RMAT) by…
The U.S. Food and Drug Administration (FDA) has selected Neurogene‘s NGN-401, an experimental gene therapy for Rett Syndrome,…