News

COVID-19 Vaccine Compatible with Trial Testing of Rett Gene Therapy

People with Rett syndrome can receive the COVID-19 vaccine from Johnson & Johnson and still participate in future clinical trials of the investigational gene therapy TSHA-102, the treatment’s developer has announced. “We are aware of concerns about the adenovirus vector COVID-19 vaccine from Johnson & Johnson (J&J) and…

Skills Training Given Before Symptoms Evident May Help Rett Children

Early, intensive training in motor and memory skills before symptoms develop may delay disease progression and help children with Rett syndrome retain specific milestones, a study in mice suggests. Observed benefits were not evident when training was given to symptomatic animals, its researchers wrote, supporting “genetic screening of…

Grant Will Fund Study of Breathing Difficulties

A $2.1 million grant will fund a study investigating if — and how — cells called astrocytes might affect the irregular breathing associated with Rett syndrome. Such disordered breathing is associated with a higher mortality rate and scientists remain unsure of how mutations in the MECP2 gene —…

Potential Gene Therapy for Rett Syndrome in the Works

A gene therapy aimed at slowing disease progression and improving movement, coordination and communication in children with Rett syndrome, is being developed. Mostly seen in girls, Rett affects brain development, causing impairments such as progressive loss of motor skills and speech. “It’s often a full-time job as a…

COVID-19 Economic Stimulus Legislation Welcomed by The Arc

The Arc and its advocates are welcoming the U.S. Congress’ decision to pass COVID-19-related economic stimulus legislation, which will extend access to Medicaid home and community-based services (HCBS) for people with intellectual and developmental disabilities (IDD). The $1.9 trillion American Rescue Plan Act of 2021, passed Tuesday and…

Taysha, Yale to Advance Gene Therapy Program

Note: This story was updated April 22, 2021, to clarify the collaboration between Yale and Taysha at the company’s request.  Taysha Gene Therapies has established a new research partnership with Yale University to advance its gene therapy program for neurodevelopmental disorders, which includes Rett syndrome, the company…