Rett Syndrome News

April 9, 2021April 9, 2021

MeCP Protein’s Ability to Interact With DNA May Be Crucial for Brain Health

Rett Syndrome News

MeCP2, the protein missing or defective in most people with Rett syndrome, contains a specific domain it can use to interact with different sets of DNA building blocks found in ... Read more

April 7, 2021April 7, 2021

LAVENDER Phase 3 Trial of Trofinetide Enrolling at New Sites

Rett Syndrome News

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 ... Read more

April 2, 2021April 2, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

Rett Syndrome News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of ... Read more

March 31, 2021March 31, 2021

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

COVID-19 News, Rett Syndrome News

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 ... Read more

March 26, 2021March 26, 2021

Skills Training Given Before Symptoms Evident May Help Rett Children

Rett Syndrome News

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 ... Read more

March 24, 2021March 24, 2021

Grant Will Fund Study of Breathing Difficulties

Rett Syndrome News

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 ... Read more

March 19, 2021March 19, 2021

Potential Gene Therapy for Rett Syndrome in the Works

Rett Syndrome News

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, ... Read more

March 17, 2021March 17, 2021

Balance of Cohesin Proteins Appear Central to X Chromosome Activity

Rett Syndrome News

A fine balance of proteins called cohesins is important for the normal inactivation of the X chromosome, new research suggests. Better understanding how the activity of the X chromosome activity ... Read more

March 12, 2021March 12, 2021

COVID-19 Economic Stimulus Legislation Welcomed by The Arc

COVID-19 News, Rett Syndrome News

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 ... Read more

March 10, 2021March 10, 2021

Taysha, Yale to Advance Gene Therapy Program

Rett Syndrome News

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. The multi-year collaboration ... Read more

March 8, 2021March 8, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

COVID-19 News, Rett Syndrome News

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 5, 2021March 5, 2021

The Arc Awarded Comcast NBCUniversal Grant for Special Education Initiative

COVID-19 News, Rett Syndrome News

A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to educational resources for students of color and economically disadvantaged youngsters with intellectual and developmental disabilities ... Read more

March 3, 2021March 3, 2021

Study Identifies Potential Blood-based Biomarkers, Paths for Treatment

Rett Syndrome News

Researchers have identified a set of RNA molecules that are dysregulated in Rett syndrome and may be relevant biomarkers for the disorder. The study, “Unraveling Molecular Pathways Altered in MeCP2-Related ... Read more

February 26, 2021February 26, 2021

Teeth Grinding Is Most Common Dental Problem

Rett Syndrome News

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 ... Read more

February 25, 2021February 25, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

COVID-19 News, Rett Syndrome News

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more