News Small Molecule Aids Motor Skills, Breathing in Rett Mouse Model

News, syndicated COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated Art Contest Salutes Creativity of Rare Disease Communities

News Girls With Rett Have Higher Rate of Menstrual Seizures, Study Says

News, syndicated NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News Distinct Immune Protein Pattern Found in Rett Patients in Small Study

News Anavex 2-73 Seen to Safely Ease Rett Severity in Women in Phase 2 Trial

News Early Work Using Lab-grown ‘Mini-brains’ Identifies 2 Potential Rett Therapies

News Anavex 2-73 Treatment for Rett to Receive New US Patent Broadening Coverage

News, syndicated NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News Reverse Rett Hosts Dec. 5 Virtual Big Night In Fundraiser, Benefitting Research

News GW Stops ARCH Phase 3 Trial Testing Epidiolex in Children, Adolescents, Due to COVID-19

News, syndicated Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News Adults With Rett Require Tailored Care Program to Manage Symptoms, Study Says

News Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties

News Taysha Gene Therapies, Catalent Partner to Advance Gene Therapies for Rett, Other Disorders

News ‘Band of Mothers’ Connect to Raise Awareness About Boys With Rett

News, syndicated NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News Rett Syndrome Research Trust Seeks Families for Global Registry

News, syndicated UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds

News, syndicated Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News FDA Acts to Support Development of Potential Gene Therapy, TSHA-102

News New KIF1A Gene Mutation Linked to Rett, Ends One Girl’s 15-year Diagnostic Journey

News, syndicated Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News Nearly $500K Awarded to Support Search for Rett Syndrome Biomarkers

News Events, Activities to Take Place Throughout October, Rett Syndrome Awareness Month

News Overground or Treadmill Walking Suitable to Assess Gait in Rett Patients, Study Finds

News, syndicated Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

News Brain Adenosine System May Be Therapeutic Target in Rett, Mouse Study Reports

News Study Reveals Similar Spinal Surgery Outcomes in Rett Syndrome and Cerebral Palsy

News Medical Advisory Board to Set Best-in-Class Care, Rettsyndrome.org Says

News Rett Patients With Milder Mutation Have Poorest Quality of Life, Study Finds

News First US-based Anavex 2-73 Trial Completed; Top-line Results Due by Year’s End

News Vagus Nerve Stimulation Plus Sound May Improve Auditory Processing in Rett, Study Suggests

News, syndicated Same But Different Photo Contest Celebrates People With Rare Diseases

News Study Links Word Acquisition With Walking Ability in Rett Syndrome

News AVXS-201, Gene Therapy for Rett, Again Moving Toward Clinical Trial, AveXis Says

News, syndicated Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News Parents of Rett Children in Spain Struggle With Healthcare Access, Time Management, Study Reports

News Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett

News, syndicated EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated New Streaming Channel Showcases Rare Disease Films

News DNA Methylation Patterns Before Birth and After May Help in Understanding Rett

News Taysha Raises $95M to Help Speed Clinical Development of Gene Therapies

News Novel Function of MeCP2 Protein Could Be Route to Rett Therapy, Study Suggests

News High Stress Levels Found in 40% of Parents Caring for Rett Child in Italy

News RNA Editing Repairs Rett Syndrome Mutation in Mouse Model

News BRD4 Protein May be Therapeutic Target for Rett Syndrome, Study Suggests

News, syndicated National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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

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