Updates

November 15, 2019November 15, 2019
FDA Grants Rare Pediatric Disease Designation to Anavex 2-73 for Rett Syndrome

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Anavex 2-73 (blarcamesine) for the treatment of Rett syndrome. This designation provides priority review ... Read more
November 15, 2019November 16, 2019
Rare Disease Film Festival Highlights Patient and Researcher Unity

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 ... Read more
November 12, 2019
New MECP2 Gene Mutations Found in Japanese Rett Syndrome Patients

Ten new mutations in the MECP2 gene have been identified in a group of children and adults with Rett syndrome, a Japanese study reports. ... Read more
November 8, 2019November 8, 2019
Animal Study Details Motor, Sensory and Respiratory Benefits of Anavex 2-73, Supporting Phase 2 Trials

Treatment with Anavex 2-73 was seen to improve motor skills, acoustic responses and visual acuity in a mouse model of Rett syndrome, supporting ongoing Phase ... Read more
November 5, 2019November 5, 2019
Newron Seeks Participants for Global Survey on Burden of Rett

In partnership with the Rett syndrome community, Newron Pharmaceuticals has launched a groundbreaking International Rett Syndrome Burden of Illness Survey. The first of its ... Read more

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Phase 3 Trial of Trofinetide Recruiting Girls and Young Women with Rett Syndrome in US
November 1, 2019November 1, 2019
A Phase 3 clinical trial is recruiting girls and young women with Rett syndrome ...
Treadmill Walking Exercises May Improve Gait Performance of Rett Syndrome Patients, Study Suggests
October 29, 2019October 29, 2019
Treadmill walking exercises can help improve gait speed and dynamics in people with Rett ...
Altered Protein Levels in Brain, Affecting Energy Metabolism, Underlies Rett Syndrome, Early Study Suggests
October 25, 2019October 25, 2019
Lack of a functional MeCP2 protein leads to Rett syndrome by altering levels of brain ...
Natural Antioxidant, CoQ10, as Dietary Supplement Eases Oxidative Stress in Rett Patients, Small Study Suggests
October 22, 2019October 22, 2019
Taking coenzyme Q10 (CoQ10) — a natural antioxidant — as an oral dietary supplement can ...
New MECP2 Mutation May Lead to Atypical Rett Syndrome, Case Study Reports
October 17, 2019October 17, 2019
A newly identified mutation in the MECP2 gene — which is linked to Rett syndrome ...
Rett Cells Have Defects in Energy Production, Greater Oxidative Stress, Study Suggests
October 15, 2019October 15, 2019
Defects in mitochondria — cells’ powerhouses — and nerve cell damage resulting from ...

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Social Media Campaigns, Purple Lighting Mark Rett Syndrome Awareness Month
October 10, 2019October 10, 2019
From social media efforts to lighting public attractions purple, efforts are underway to observe ...
‘Uptime’ Physical Activities Aid Life Quality and Walking Abilities in Rett Patients, Study Finds
October 8, 2019October 8, 2019
“Uptime” activities, such as standing and walking, that promote physical activity and work against a sedentary ...
Resting Heart Rate Variability May Predict Reactivity to Tactile Stimuli, Study Suggests
October 3, 2019October 3, 2019
Reactivating Silenced X Chromosome Could Eventually Lead to Targeted Therapies, Researchers Say
October 1, 2019October 1, 2019
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
September 27, 2019September 26, 2019
Rettsyndrome.org’s New CSO Has a Personal Connection to Disease
September 26, 2019September 26, 2019

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Rare Case of Brothers with Rett Suggests Multiple Factors in Disease Course
September 24, 2019September 24, 2019
Two brothers with Rett syndrome carrying the same MECP2 mutation display distinct disease courses, a ...
Preliminary Data From Anavex-2-73 Phase 2 Trial Suggest Safety, Efficacy
September 19, 2019September 19, 2019
Sensory Therapy Can Improve Hand Movements in Kids with Rett, Study Suggests
September 17, 2019September 17, 2019
EXCELLENCE Trial Will Test Anavex-2-73 in Children, Adolescents
September 10, 2019September 10, 2019

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Existing Compounds Can Target KCC2 Protein to Ease Rett Symptoms, Early Study Reports
September 5, 2019September 5, 2019
Several compounds already approved by the U.S. Food and Drug Administration (FDA) can increase ...
X Chromosome Inactivation Not the Only Contributing Factor for Disease Severity in Female Rett Patients, Study Contends
September 3, 2019September 3, 2019
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
September 2, 2019August 30, 2019

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AveXis Delays Trial of Rett Gene Therapy, AVXS-201, to Conduct New Tests and Quality Control Checks
August 29, 2019August 29, 2019
AveXis is postponing plans to open a clinical trial of its Rett syndrome gene therapy ...
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
August 29, 2019August 28, 2019
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is ...
Chemical Modifications That Accelerate KCC2 Activity May Ease Symptoms of Rett Syndrome, Mouse Study Indicates
August 27, 2019August 27, 2019
Chemical modifications in the protein KCC2 that accelerate its activity during brain development may ...
Monkeys With Rett Pay More Attention to Stronger Social Visual Stimuli, Study Finds
August 22, 2019August 22, 2019
Monkeys with Rett syndrome pay more attention to stronger social visual stimuli than healthy ...
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
August 20, 2019
Imagine living your whole life with a painful disease so rare that only 25 ...
Meta-Analysis of Genetic Studies Essential to Discover New Therapeutic Targets for Rett Syndrome, Reviewers Contend
August 13, 2019August 13, 2019
Implementation of meta-analysis — a statistical approach that combines information from different studies — ...
Algorithm to Measure Pupil Dilation, Heart Rate Could Help Detect Rett Earlier
August 8, 2019August 8, 2019
An algorithm that measures spontaneous variations in pupil dilation or heart rate could allow ...
European Agency Recommends Orphan Designation for Anavex 2-73
August 6, 2019August 6, 2019
The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has endorsed Anavex 2-73 ...
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
August 1, 2019
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, ...

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