News First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett

News New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome

News Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model

cystic fibrosis, News, syndicated Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

News US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal

News Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US

News Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy

News Coalition Will Address Racial Disparities in Rare Disease Communities

News Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett

News, syndicated COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News Blocking P2X7 Receptors May Be Therapeutic Target for Rett, Mouse Study Shows

News, syndicated Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News Scientists Who Discovered MECP2 Link with Rett Syndrome Awarded 2020 Brain Prize

cystic fibrosis, News, syndicated Video Games Connect Chronically Ill Children Isolated at Home, Hospital

News, syndicated Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders

News, syndicated Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News Newron Terminates Sarizotan Development Due to STARS Trial Failure

News, syndicated Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

News Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model

News, syndicated NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News Altered Nerve Cell Communication Tied to Motor Difficulties of Rett

News Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment

News NORD’s Advice on COVID-19: You’re Stronger Than You Think

News Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits

News NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome

Just Published, News Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

News Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon

News Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds

News FDA Grants Rare Pediatric Disease Designation to Trofinetide for Rett Syndrome

News Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News Restoring Wnt6 Protein Eases Behavioral, Motor Impairment, Mouse Study Suggests

News Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

News Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

News Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News Newron Expands Rett Burden of Illness Survey to Europe and Australia

News Girls with Rett Need Better Speech Therapy and Communication Support in Schools, Survey Says

News FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News FDA Grants Fast Track Designation to Anavex 2-73 for Treatment of Rett Syndrome

News MECP2 Gene Mutations Alter Nerve Cells and Impair Learning, Mouse Study Finds

News Scientists Identify 2 New Genetic Mutations in 2 Iranian Girls With Rett Syndrome

News Study Suggests Critical Time Window to Treat Rett Syndrome

News Modification in Huntingtin Protein Prolongs Survival, Improves Lung Function in Rett Mouse Model, Study Finds

News DNA Sequencing Identifies Unusual Genetic Cause of Rett-like Disease in Young Girl

News Mutations in Only One MeCP2 Protein Form Leads to Milder Rett Symptoms in Boy, Report Indicates

News Anavex 2-73 Earns U.S. Patent for Neurodevelopmental Disorders, Including Rett Syndrome

News Low KCC2 Protein Levels May Underlie Developmental Defects in Rett Patients, Study Suggests

News New Genetic Causes of Rett Syndrome Found in Chinese Infants, Study Reports

News Stemonix and Atomwise Join Forces to Develop Small Molecules Targeting Rett Syndrome

News Parents of Rett Patients View Deep Brain Stimulation as Viable Option

News FDA, Newron Pharma to Meet in Step to Releasing Results of Sarizotan Pivotal Trial

News Gene Therapy Takes Center Stage at 2019 NORD Summit

News Altered Gut Microbiota Could Be Therapeutic Target in Rett Syndrome, Study Suggests

News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News Sarepta and StrideBio Partner to Advance Gene Therapies for Rett Syndrome, Other Disorders

News FDA Grants Rare Pediatric Disease Designation to Sarizotan for Treating Rett Syndrome

News Abnormal Breakdown of Certain Lipids May Underlie Rett Syndrome Development, Study Suggests

News FDA Grants Rare Pediatric Disease Designation to Anavex 2-73 for Rett Syndrome

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News New MECP2 Gene Mutations Found in Japanese Rett Syndrome Patients

News Animal Study Details Motor, Sensory and Respiratory Benefits of Anavex 2-73, Supporting Phase 2 Trials

News Newron Seeks Participants for Global Survey on Burden of Rett

News Phase 3 Trial of Trofinetide Recruiting Girls and Young Women with Rett Syndrome in US

News Treadmill Walking Exercises May Improve Gait Performance of Rett Syndrome Patients, Study Suggests

News Altered Protein Levels in Brain, Affecting Energy Metabolism, Underlies Rett Syndrome, Early Study Suggests

News Natural Antioxidant, CoQ10, as Dietary Supplement Eases Oxidative Stress in Rett Patients, Small Study Suggests

News New MECP2 Mutation May Lead to Atypical Rett Syndrome, Case Study Reports

News Rett Cells Have Defects in Energy Production, Greater Oxidative Stress, Study Suggests

News Social Media Campaigns, Purple Lighting Mark Rett Syndrome Awareness Month

News ‘Uptime’ Physical Activities Aid Life Quality and Walking Abilities in Rett Patients, Study Finds

News Resting Heart Rate Variability May Predict Reactivity to Tactile Stimuli, Study Suggests

News Reactivating Silenced X Chromosome Could Eventually Lead to Targeted Therapies, Researchers Say

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News Rettsyndrome.org’s New CSO Has a Personal Connection to Disease

News Rare Case of Brothers with Rett Suggests Multiple Factors in Disease Course

News Preliminary Data From Anavex-2-73 Phase 2 Trial Suggest Safety, Efficacy

News Sensory Therapy Can Improve Hand Movements in Kids with Rett, Study Suggests

News EXCELLENCE Trial Will Test Anavex-2-73 in Children, Adolescents

News Existing Compounds Can Target KCC2 Protein to Ease Rett Symptoms, Early Study Reports

News X Chromosome Inactivation Not the Only Contributing Factor for Disease Severity in Female Rett Patients, Study Contends

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News AveXis Delays Trial of Rett Gene Therapy, AVXS-201, to Conduct New Tests and Quality Control Checks

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News Chemical Modifications That Accelerate KCC2 Activity May Ease Symptoms of Rett Syndrome, Mouse Study Indicates

News Monkeys With Rett Pay More Attention to Stronger Social Visual Stimuli, Study Finds

News Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News Meta-Analysis of Genetic Studies Essential to Discover New Therapeutic Targets for Rett Syndrome, Reviewers Contend

News Algorithm to Measure Pupil Dilation, Heart Rate Could Help Detect Rett Earlier

News European Agency Recommends Orphan Designation for Anavex 2-73

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News Thyroid Hormone Imbalance May Contribute to Rett Syndrome, Study Suggests

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News Microswitch Intervention Program May Benefit Children with Rett Syndrome, Study Suggests

News Exosomes May Have Therapeutic Use in Rett Syndrome, Study Finds

News microRNAs Could Serve as Biomarkers of Rett’s Course, Study Suggests

News European Initiative Targets Diagnosis, Treatment of Rare Diseases