Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
Articles by Marisa Wexler, MS
Early, intensive training in motor and memory skills before symptoms develop may delay disease progression and help children with…
A fine balance of proteins called cohesins is important for the normal inactivation of the X chromosome, new research suggests.
Researchers have identified a set of RNA molecules that are dysregulated in Rett syndrome and may be relevant biomarkers…
Novartis Gene Therapies is planning to submit an investigational new drug application (IND) for OAV201, its Rett syndrome gene…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
A Phase 2 clinical trial evaluating the safety and efficacy of Anavex Life Sciences‘ investigational therapy Anavex…
Stimulation of the vagus nerve, when paired with sound, could help people with Rett syndrome better process what they…
Children with Rett syndrome who can verbalize meaningful words are more likely to be able to walk past age…