The Ontario Rett Syndrome Association (ORSA) announced the awarding of two grants to support research into Rett syndrome in…
Articles by Marisa Wexler, MS
A music-based intervention helped to normalize certain social behaviors in a mouse model of Rett syndrome, researchers reported. Findings also…
Alcyone Therapeutics, a new company focused on developing gene therapies for disorders that affect the nervous system, announced its…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
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…