Rett Syndrome News

October 23, 2020October 23, 2020

Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds

Rett Syndrome News

Treatment with the antidepressant mirtazapine preserves motor and respiratory function, as well as social behavior, in women with Rett syndrome, a study shows. Improved motor function also was seen in ... Read more

October 16, 2020October 16, 2020

FDA Acts to Support Development of Potential Gene Therapy, TSHA-102

Rett Syndrome News

The U.S. Food and Drug Administration (FDA) has granted orphan drug and rare pediatric disease designations to TSHA-102, a potential gene therapy for Rett syndrome. Taysha Gene Therapies, the treatment’s developer, plans ... Read more

October 14, 2020October 14, 2020

New KIF1A Gene Mutation Linked to Rett, Ends One Girl’s 15-year Diagnostic Journey

Rett Syndrome News

Researchers have identified a new mutation in the KIF1A gene in a girl in Australia that is associated with Rett syndrome, ending her 15-year diagnostic journey. Two new other KIF1A mutations ... Read more

October 7, 2020October 7, 2020

Nearly $500K Awarded to Support Search for Rett Syndrome Biomarkers

Rett Syndrome News

The National Institute for Neurological Disorders and Stroke, a division of the National Institutes of Health (NIH), awarded nearly $500,000 for a study seeking biomarkers of Rett syndrome. The $498,572 grant was ... Read more

October 2, 2020October 2, 2020

Events, Activities to Take Place Throughout October, Rett Syndrome Awareness Month

Rett Syndrome News

A number of events are in the works to mark Rett Syndrome Awareness Month, observed each October to heighten awareness about the rare neurodevelopmental disorder. Patients, caregivers, and advocates may, ... Read more

September 30, 2020September 30, 2020

Overground or Treadmill Walking Suitable to Assess Gait in Rett Patients, Study Finds

Rett Syndrome News

Walking overground or on a treadmill is suitable as a tool to observe and detect gait changes in people with Rett syndrome, a small study has found. The study, “Characteristic ... Read more

September 25, 2020September 25, 2020

Brain Adenosine System May Be Therapeutic Target in Rett, Mouse Study Reports

Rett Syndrome News

Addressing changes in the adenosine system, essential for nerve cell function in the brain, may be a therapeutic strategy in Rett syndrome, a study in mice reported. The study, “Impairment ... Read more

September 23, 2020September 23, 2020

Study Reveals Similar Spinal Surgery Outcomes in Rett Syndrome and Cerebral Palsy

Rett Syndrome News

Patients with Rett syndrome have similar spinal surgery outcomes to those of people with cerebral palsy (CP), according to new research. The findings also revealed comparable degrees of spine abnormalities ... Read more

September 18, 2020September 21, 2020

Medical Advisory Board to Set Best-in-Class Care, Rettsyndrome.org Says

Rett Syndrome News

Rettsyndrome.org has launched the International Rett Syndrome Foundation Medical Advisory Board, aiming to enhance care and research to help people with the neurodevelopmental disorder, according to the non-profit. The Advisory ... Read more

September 16, 2020September 16, 2020

Rett Patients With Milder Mutation Have Poorest Quality of Life, Study Finds

Rett Syndrome News

Girls and women with Rett syndrome caused by a mutation usually associated with milder disease have the poorest quality of life, according to a large study. Data from this and ... Read more

September 11, 2020September 11, 2020

First US-based Anavex 2-73 Trial Completed; Top-line Results Due by Year’s End

COVID-19 News, Rett Syndrome News

A Phase 2 clinical trial evaluating the safety and efficacy of Anavex Life Sciences‘ investigational therapy Anavex 2-73 (blarcamesine) in Rett syndrome has been completed, according to the company. Top-line ... Read more

September 9, 2020September 9, 2020

Vagus Nerve Stimulation Plus Sound May Improve Auditory Processing in Rett, Study Suggests

Rett Syndrome News

Stimulation of the vagus nerve, when paired with sound, could help people with Rett syndrome better process what they hear, a study in rats suggests. The study, “Vagus nerve stimulation ... Read more

September 2, 2020September 3, 2020

Study Links Word Acquisition With Walking Ability in Rett Syndrome

Rett Syndrome News

Children with Rett syndrome who can verbalize meaningful words are more likely to be able to walk past age 10, a study suggests. The study, “Meaningful word acquisition is associated ... Read more

August 28, 2020August 28, 2020

AVXS-201, Gene Therapy for Rett, Again Moving Toward Clinical Trial, AveXis Says

Rett Syndrome News

Most animal studies needed to support plans to move AVXS-201 — AveXis’ investigational gene therapy for Rett syndrome — into clinical testing in patients are complete, the company reports. AveXis hopes to ... Read more

August 27, 2020September 28, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Rett Syndrome News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more