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Overground or Treadmill Walking Suitable to Assess Gait in Rett Patients, Study Finds
Walking overground or on a treadmill is suitable as a tool to observe and detect ...
More from Rett Syndrome News
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Brain Adenosine System May Be Therapeutic Target in Rett, Mouse Study Reports
Addressing changes in the adenosine system, essential for nerve cell function in the brain, may ... -
Study Reveals Similar Spinal Surgery Outcomes in Rett Syndrome and Cerebral Palsy
Patients with Rett syndrome have similar spinal surgery outcomes to those of people with cerebral ... -
Medical Advisory Board to Set Best-in-Class Care, Rettsyndrome.org Says
Rettsyndrome.org has launched the International Rett Syndrome Foundation Medical Advisory Board, aiming to enhance care ... -
Rett Patients With Milder Mutation Have Poorest Quality of Life, Study Finds
Girls and women with Rett syndrome caused by a mutation usually associated with milder disease ... -
First US-based Anavex 2-73 Trial Completed; Top-line Results Due by Year’s End
A Phase 2 clinical trial evaluating the safety and efficacy of Anavex Life Sciences‘ investigational ... -
Vagus Nerve Stimulation Plus Sound May Improve Auditory Processing in Rett, Study Suggests
Stimulation of the vagus nerve, when paired with sound, could help people with Rett syndrome ... -
Same But Different Photo Contest Celebrates People With Rare Diseases
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is ... -
Study Links Word Acquisition With Walking Ability in Rett Syndrome
Children with Rett syndrome who can verbalize meaningful words are more likely to be able ... -
AVXS-201, Gene Therapy for Rett, Again Moving Toward Clinical Trial, AveXis Says
Most animal studies needed to support plans to move AVXS-201 — AveXis’ investigational gene therapy for ... -
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ... -
Parents of Rett Children in Spain Struggle With Healthcare Access, Time Management, Study Reports
Parents of children with Rett syndrome face difficulties with healthcare access, time management, bureaucracy, and ... -
Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett
To better understand X-linked disorders such as Rett syndrome, researchers discovered an enzyme called DCP1A ... -
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ... -
New Streaming Channel Showcases Rare Disease Films
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated ... -
DNA Methylation Patterns Before Birth and After May Help in Understanding Rett
Researchers at the Salk Institute for Biological Studies have mapped how DNA methylation — a ... -
Taysha Raises $95M to Help Speed Clinical Development of Gene Therapies
Taysha Gene Therapies received $95 million in funding that will support the clinical development of ... -
Novel Function of MeCP2 Protein Could Be Route to Rett Therapy
Mutations in the MECP2 gene, which lead to most cases of Rett syndrome, prevent the ... -
High Stress Levels Found in 40% of Parents Caring for Rett Child in Italy
About 40% of parents whose daughters have Rett syndrome show clinically high levels of stress, ... -
RNA Editing Repairs Rett Syndrome Mutation in Mouse Model
For the first time, scientists have repaired a mutation in mice that underlies Rett syndrome, ... -
BRD4 Protein May be Therapeutic Target for Rett Syndrome, Study Suggests
By targeting a protein called BRD4, an experimental cancer treatment known as JQ1 reversed cellular abnormalities ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ... -
First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett
A Phase 2/3 trial evaluating the treatment candidate Anavex-2-73 (blarcamesine) in children and adolescents with Rett ... -
New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome
Researchers have discovered that early inactivation of the X chromosome is a two-step process regulated ... -
Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model
Treatment with the diabetes therapy metformin is able to restore energy production in the brain ... -
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those ... -
US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal
U.S. enrollment for a Phase 2 clinical trial of investigational therapy Anavex 2-73 (blarcamesine) for Rett ... -
Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US
Acadia Pharmaceuticals is again enrolling in its Phase 3 LAVENDER trial testing trofinetide as a treatment ... -
Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy
Combining conventional cognitive training with transcranial direct current stimulation (tDCS) — a non-invasive, painless brain ... -
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on ... -
Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett
Several genes involved in lipid (fat) metabolism, nerve cell communication, and DNA damage response may ... -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for ... -
Blocking P2X7 Receptors May Be Therapeutic Target for Rett
Blocking the activity of pro-inflammatory P2X7 receptors — a class of receptors found in immune ... -
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
European authorities must step up efforts to screen babies for a multitude of genetic disorders, ... -
Scientists Who Discovered Gene Link with Rett Syndrome Awarded 2020 Brain Prize
The scientists who discovered the MECP2 gene and its role in Rett syndrome have been ... -
Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders
Taysha Gene Therapies has raised $30 million and established a partnership with UT Southwestern Medical ... -
Newron Terminates Sarizotan Development Due to STARS Trial Failure
Treatment with sarizotan failed to reduce apnea episodes (breathing stops) while awake compared to a ... -
Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model
A form of gene therapy based on providing an unstable version of the MECP2 gene ... -
Altered Nerve Cell Communication Tied to Motor Difficulties of Rett
Problems with nerve cell communication and adaptation in the brain’s motor cortex is associated with ... -
Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment
A comprehensive metabolic analysis uncovered significantly different levels of 66 molecules in people with Rett ... -
NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with ... -
Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits
Anavex Life Sciences announced plans to continue to screen and enroll patients into its active ... -
NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with ... -
Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome
Treatment with insulin-like growth factor 1 (IGF-1) eases symptoms of Rett syndrome by suppressing the ... -
Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases
Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will ... -
Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon
Newron Pharmaceuticals has received the final minutes from a meeting with the U.S. Food and ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according ... -
MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds
The type of mutation in the MECP2 gene in people with Rett syndrome is a ... -
FDA Grants Rare Pediatric Disease Designation to Trofinetide for Rett Syndrome
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to trofinetide, a potential ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign ... -
Restoring Wnt6 Protein Eases Behavioral, Motor Impairment, Mouse Study Suggests
Restoring the levels of Wnt6, a signaling molecule involved in brain function, eased motor and behavioral ... -
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles ... -
Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave ... -
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 ...