A gene therapy-like approach that provided a full-length MECP2 — the gene mutated in most cases of Rett syndrome…
Articles by Marta Figueiredo, PhD
TSHA-102, an experimental gene therapy for Rett syndrome, safely and effectively normalized levels of MeCP2 — the protein missing…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
People with Rett syndrome have significantly weaker electrical responses in the brain to visual and auditory stimuli than do…
Scientists have identified two compounds that were able to rescue nerve cell defects caused by mutations in the MECP2 gene…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
Researchers have identified a new mutation in the KIF1A gene in a girl in Australia that is associated with…
Girls and women with Rett syndrome caused by a mutation usually associated with milder disease have the poorest quality…
Combining conventional cognitive training with transcranial direct current stimulation (tDCS) — a non-invasive, painless brain stimulation approach — safely…
Several genes involved in lipid (fat) metabolism, nerve cell communication, and DNA damage response may be therapeutic targets for…