Patricia Inácio, PhD, science writer —

The International Rett Syndrome Foundation (IRSF) has designated 15 clinics across the U.S. as members of the Center of Excellence Network. These 15 centers, some newly designated and located in 14 states across the country, will integrate the foundation’s network of clinics designed to provide best-in-class care for people…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Acadia Pharmaceuticals is enrolling participants at four new clinical sites in its Phase 3 LAVENDER trial testing trofinetide as a treatment for girls and young women with Rett syndrome. In an online letter to the Rett community, Acadia announced these recent additions: Seattle Children’s Hospital; the University of North Carolina…

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 signed…

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 announced.

Bruxism, also known as teeth grinding and jaw clenching, is the most common oral manifestation in patients with Rett syndrome, according to a small review of literature. The study, “Oral Manifestations of Rett Syndrome—A Systematic Review,” was published in the International Journal of Environmental Research and Public…

Girls and women with Rett syndrome may have increased rate of menstrual seizures, also known as catamenial seizures, compared to healthy females, a study reports. Age of onset, and duration and flow of menstruation in girls and women with Rett were similar to healthy girls. Bone health concerns was…

The transition into adulthood of women with Rett syndrome warrants a tailored program and a multidisciplinary team to provide the best clinical care, according to a 13-year Italian study of the complex symptoms experienced by these patients. Most adults with Rett deal with symptoms ranging from epilepsy and bone…

Taysha Gene Therapies and Catalent have established a partnership to advance Taysha’s gene therapies for neurological disorders, including Rett syndrome. The partnership allows Taysha to use Catalent’s gene therapy facilities in Maryland to expand the company’s manufacturing capacity. The gene therapy for Rett, called TSHA-102, uses a modified,…