Rett Syndrome News

January 15, 2021January 15, 2021

Girls With Rett Have Higher Rate of Menstrual Seizures, Study Says

Rett Syndrome News

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 ... Read more

January 8, 2021January 8, 2021

Distinct Immune Protein Pattern Found in Rett Patients in Small Study

Rett Syndrome News

In people with Rett syndrome, cells known as fibroblasts have a distinct protein production profile — called a proteome — that overrepresents biological pathways related to immune function and inflammation, a ... Read more

December 18, 2020December 18, 2020

Anavex 2-73 Seen to Safely Ease Rett Severity in Women in Phase 2 Trial

Rett Syndrome News

Anavex 2-73 (blarcamesine), an investigational treatment for Rett syndrome, lessened the disease’s characteristic behavioral features, its severity, and levels of the glutamate biomarker with no serious side effects, top-line results of ... Read more

December 11, 2020December 11, 2020

Early Work Using Lab-grown ‘Mini-brains’ Identifies 2 Potential Rett Therapies

Rett Syndrome News

Scientists have identified two compounds that were able to rescue nerve cell defects caused by mutations in the MECP2 gene — the most common cause of Rett syndrome — in ... Read more

December 4, 2020December 4, 2020

Anavex 2-73 Treatment for Rett to Receive New US Patent Broadening Coverage

Rett Syndrome News

Anavex Life Sciences has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for a patent application covering Anavex 2-73 (blarcamesine) and its related sigma-1 receptor ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

COVID-19 News, Rett Syndrome News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 25, 2020November 25, 2020

Reverse Rett Hosts Dec. 5 Virtual Big Night In Fundraiser, Benefitting Research

Rett Syndrome News

Viewers from around the world are invited to join in the virtual Reverse Rett Big Night In, a U.K. fundraiser benefitting Rett syndrome research, to be held Saturday, Dec. 5 ... Read more

November 20, 2020November 20, 2020

GW Stops ARCH Phase 3 Trial Testing Epidiolex in Children, Adolescents, Due to COVID-19

COVID-19 News, Rett Syndrome News

Due to the unprecedented challenges caused by the ongoing COVID-19 pandemic, GW Pharmaceutics has stopped the ARCH Phase 3 clinical trial evaluating the oral cannabidiol (CBD) solution Epidiolex in children and adolescents ... Read more

November 18, 2020November 18, 2020

Adults With Rett Require Tailored Care Program to Manage Symptoms, Study Says

Rett Syndrome News

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 ... Read more

November 13, 2020November 13, 2020

Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties

Rett Syndrome News

Problems with skilled learning tasks that involve motor function and making sense of sounds — known to be affected by Rett syndrome — were seen in rat model of the disease ... Read more

November 11, 2020November 11, 2020

Taysha Gene Therapies, Catalent Partner to Advance Gene Therapies for Rett, Other Disorders

Rett Syndrome News

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 ... Read more

November 6, 2020November 6, 2020

‘Band of Mothers’ Connect to Raise Awareness About Boys With Rett

Rett Syndrome News

Of the people who know about Rett syndrome, many think it affects only females. But families of boys with this disorder know differently. Parents of children with an ultra-rare manifestation of ... Read more

October 30, 2020October 30, 2020

Rett Syndrome Research Trust Seeks Families for Global Registry

Rett Syndrome News

As the Rett Syndrome Research Trust (RSRT) prepares to launch a global database of caregiver-provided information called The Rett Syndrome Global Registry, it is seeking families to participate in a ... Read more

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