Rett Syndrome News

October 15, 2021October 15, 2021

Communication Interventions May Help Rett Patients Better Interact

Rett Syndrome News

A comprehensive communication intervention package improved the expressive communication and/or visual attention of three women with Rett syndrome, a small study shows. These findings suggest that the intervention — which combined ... Read more

October 13, 2021October 13, 2021

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

COVID-19 News, Rett Syndrome News

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC ... Read more

October 8, 2021October 8, 2021

Phase 2/3 Trial of Anavex 2-73 Enrolling Girls in Canada, Australia

Rett Syndrome News

A clinical trial evaluating oral Anavex 2-73 in children and teenagers with Rett syndrome is recruiting up to 84 girls, ages 5 to 17, at a center in Vancouver, Canada, and three ... Read more

October 6, 2021October 6, 2021

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

Rett Syndrome News

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and ... Read more

October 1, 2021October 1, 2021

It’s October: Are You Ready for Rett Awareness?

Rett Syndrome News

Supporters worldwide are marking Rett Syndrome Awareness Month, set aside each October to bring attention to Rett syndrome, a rare neurodevelopmental disorder that’s estimated to affect 1 of every 10,000 ... Read more

September 29, 2021September 29, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Rett Syndrome News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 24, 2021September 24, 2021

Experimental Rett Therapy Now an Orphan Drug in Europe

Rett Syndrome News

The European Commission has granted orphan drug designation to TSHA-102, an investigational gene therapy for the treatment of Rett syndrome. The designation encourages the development of medicines to diagnose, prevent, ... Read more

September 22, 2021September 22, 2021

Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness

COVID-19 News, Rett Syndrome News

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games ... Read more

September 17, 2021September 17, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

Rett Syndrome News

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 15, 2021September 15, 2021

IRSF Joins Standard of Excellence Program

Rett Syndrome News

The International Rett Syndrome Foundation (IRSF), an organization focused on accelerating research to treat and cure Rett syndrome, has been appointed to the National Health Council (NHC) Standard of Excellence ... Read more

September 10, 2021September 10, 2021

Inhibitory Signals in Brain May Underlie Breathing Problems in Rett

Rett Syndrome News

Abnormally slow or shallow breathing in Rett syndrome develops in part due to altered inhibitory signals in the brain, research done in a rat model of the disease suggests. Data ... Read more

September 10, 2021September 10, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Rett Syndrome News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 8, 2021September 8, 2021

Rett Patients Can Engage With, Benefit From Online Schooling

COVID-19 News, Rett Syndrome News

Participating in school virtually — using a computer with a webcam — is socially and cognitively engaging for people with Rett syndrome, according to a study in Italy. This finding ... Read more

September 3, 2021September 3, 2021

Acadia Opening Phase 2/3 Trial of Trofinetide in Young Girls, 2-5

COVID-19 News, Rett Syndrome News

Acadia Pharmaceuticals is launching an open-label clinical trial of its oral investigational medication trofinetide in toddlers and young girls with Rett syndrome, the company announced in a letter to the Rett community. ... Read more

September 2, 2021September 2, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Rett Syndrome News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more