A girl with Rett syndrome participating in a clinical trial testing gene therapy NGN-401 experienced a serious, treatment-related immune…
Articles by Steve Bryson, PhD
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,…
MeCP2, the protein impaired in most cases of Rett syndrome, controls the production of alpha-synuclein, a protein implicated in…
The Rett Syndrome Research Trust (RSRT) has partnered with Vivalink to use its Multi-Vital ECG wearable patch…
New research supports how the lack of Mecp2 protein impairs the function of healthy nerve cells in a mouse study…
A lack of MECP2 protein, the chief underlying cause of Rett syndrome, prevented precursor cells from transforming into fully…
The International Rett Syndrome Foundation (IRSF) has launched the My Rett Ally app as a digital tool to help…