Rett Syndrome News

July 28, 2021July 28, 2021

Virginia Tech Student Awarded NIH Grant for Research Into Rett Protein

Rett Syndrome News

A first-generation university student has been awarded a six-year, $466,669 grant from the National Institutes of Health (NIH) to fund his doctoral and postdoctoral research into brain-derived neurotrophic factor (BDNF), ... Read more

July 23, 2021July 23, 2021

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

Rett Syndrome News

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies ... Read more

July 21, 2021July 21, 2021

Shape Therapeutics Raises $112M to Develop RNA-based Gene Therapies

COVID-19 News, Rett Syndrome News

Shape Therapeutics has raised $112 million in financing to support work into developing its portfolio of RNA technologies to potentially treat genetic diseases, including Rett syndrome. “We have witnessed the ... Read more

July 16, 2021July 16, 2021

Nominations Open for 2022 Eurordis Black Pearl Awards

Rett Syndrome News

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work ... Read more

July 14, 2021July 14, 2021

Rett Researcher Discovers Molecular Mechanism of MECP2 Binding

COVID-19 News, Rett Syndrome News

The molecular mechanism by which the protein MeCP2 recognizes and binds to CA repeats — repetitive short sequences of DNA that are found throughout the genome — has been unraveled ... Read more

July 9, 2021July 9, 2021

Mouse Study Suggests Need to Test Therapeutic Approaches Across Rett Models

Rett Syndrome News

Treatment with a protein called CNF1 produced different results in two different mouse models of Rett syndrome, demonstrating a need to test therapeutic approaches across disease models, according to a ... Read more

July 7, 2021July 7, 2021

Virtual Avatar Improved Attention and Memory

COVID-19 News, Rett Syndrome News

Using a virtual avatar to guide users during attention and memory tasks significantly improves the performance of people with Rett syndrome, according to a small study from Italy. The human-like interactive ... Read more

July 2, 2021July 2, 2021

MeCP2 Protein’s Lack May Be Driver of Neuroinflammation in Patients

Rett Syndrome News

The lack of a working MeCP2 protein — the most common cause of Rett syndrome — skews the immune system toward a proinflammatory state in the central nervous system (CNS, ... Read more

July 1, 2021July 1, 2021

Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years

Rett Syndrome News

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, ... Read more

June 30, 2021June 30, 2021

MicroRNAs Improve Behavior, Motor Function in Rett Mouse Model

Rett Syndrome News

The use of microRNA molecules that targeted a protein receptor important for nerve cell growth led to improved behaviors and motor function in a Rett syndrome mouse model, a study ... Read more

June 25, 2021June 25, 2021

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

Rett Syndrome News

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health ... Read more

June 23, 2021June 23, 2021

Latest Trial Results of Anavex 2-73 ‘Very Promising’

Rett Syndrome News

Clinical improvements with Anavex 2-73 (blarcamesine) correlated with activation of the SIGMAR1 gene, which provides instructions for making the protein targeted by the investigational Rett syndrome therapy, in a Phase 2 study. ... Read more

June 18, 2021June 18, 2021

Caregivers Asked to Help in Adapting Tool ‘Critical’ to Therapy Trials

Rett Syndrome News

The nonprofit Rett Syndrome Research Trust (RSRT) and Duke University’s Center for Health Measurement are asking Rett syndrome caregivers to help with a study aiming to adapt and validate a tool that could ... Read more

June 16, 2021June 16, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

COVID-19 News, Rett Syndrome News

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 11, 2021June 11, 2021

Alcyone Therapeutics Opens, Focus on Gene Therapy for Rett Syndrome

Rett Syndrome News

Alcyone Therapeutics, a new company focused on developing gene therapies for disorders that affect the nervous system, announced its official launch. One of its lead candidates, ACTX-101, is an investigational ... Read more