Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Newron Expands Rett Burden of Illness Survey to Europe and Australia

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

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

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

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

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

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

Study Suggests Critical Time Window to Treat Rett Syndrome

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

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

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

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

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

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

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

Parents of Rett Patients View Deep Brain Stimulation as Viable Option

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

Gene Therapy Takes Center Stage at 2019 NORD Summit

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

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

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

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

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

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

Rare Disease Film Festival Highlights Patient and Researcher Unity

New MECP2 Gene Mutations Found in Japanese Rett Syndrome Patients

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

Newron Seeks Participants for Global Survey on Burden of Rett

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

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

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

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

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

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

Social Media Campaigns, Purple Lighting Mark Rett Syndrome Awareness Month

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

European Agency Recommends Orphan Designation for Anavex 2-73

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

Thyroid Hormone Imbalance May Contribute to Rett Syndrome, Study Suggests

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

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

Exosomes May Have Therapeutic Use in Rett Syndrome, Study Finds

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

European Initiative Targets Diagnosis, Treatment of Rare Diseases

Neurological Changes Present at Birth in Rett Syndrome, Mouse Study Finds

Measures of Quality of Life Used for Children with Rett Also Valuable During Adulthood, Study Suggests

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

First Patient Dosed in Phase 2 Extension Study of Anavex 2-73

Subtle Signs of Rett Could Be Opportunity for Early Intervention, Study Says

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

L-serine Improved Outcomes in Girl with Atypical Rett Syndrome, Case Report Says

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

Phase 2 AVATAR Study Testing Anavex 2–73 for Rett Syndrome Doses First Patient in Australia

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

MeCP2 Optimized for Brain Delivery Holds Promise as Protein Replacement Therapy for RTT, Early Study Suggests

Rettsyndrome.org Funds Research of Rett and MECP2 Syndromes

Stem Cells of Rett Mice Prone to Arrested Growth, Less Able to Produce Energy, Study Shows

Cannabidivarin Rescues Memory Deficits in Mouse Model of Rett, Study Shows

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

Teeth Misalignment Linked to Swallowing Difficulties in Girls with Rett Syndrome, Study Says

New Phase 2 Study on Anavex 2-73 for Rett Syndrome Begins in Australia

#AANAM – Experimental Gene Therapy AVXS-201 Shows Promise to Treat Rett Syndrome in Preclinical Studies

Overabundance of Nerve Fibers in the Skin May Affect Pain Sensitivity, Study Suggests

Study in Rats Provides Clues to Nerve Cell Connectivity and Body Movement Control

Mutations Causing Rett Syndrome Lead to Changes in Several Genes, Study Finds

‘Minds Wide Open’ Documentary Highlights Advances in Brain Research

Early Protein Alterations May Drive the Development of Rett Biomarkers

Physical Therapy and Other Interventions May Prevent Motor Deterioration in Patients with Rett Syndrome, Case Report Finds

Phase 3 Trial for Trofinetide Planned After Positive Phase 2 Results in Rett Syndrome

Better Preclinical Practices Would Boost Success in Clinical Setting for Rett Syndrome Therapies, Review Says

Parents of Girls with Rett Syndrome Need More Information About Treatment, Study Says

Annual Zoledronic Acid Injection May Improve Bone Health in Children with Rett Syndrome

Phase 2 Trial Evaluating Anavex 2-73 for Rett Syndrome Doses First Patient

Mosaic Mutations May Hold Key to Understanding Rett’s Development

Study Reports Rising Trend Of Prenatal Genetic Tests For Rett Syndrome

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

Understanding of How Females Inherit Traits May Hold Key to Treatment of X-linked Disorders

Misregulation in NPCs’ Development Might be Implicated in Rett Syndrome, Study Says