Updates
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News, syndicated Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
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News Cerebellum Dysfunction May Contribute to Rett Motor Problems
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News, syndicated Rare Disease Day at NIH, Set for March 1, Growing Year by Year
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News DiamiR Receives Patent Allowance for Rett MicroRNA Biomarker
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News, syndicated Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
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News Novartis to Seek Approval by Year’s End to Test Gene Therapy Candidate
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News, syndicated Pandemic Won’t Stop Rare Disease Day on Feb. 28
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News Electrical Responses in Brain to Stimuli May Be Marker of Rett Severity
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News, syndicated NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
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News Iron Deposits in Brain May Contribute to Dystonia in Rett Patients
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News IQSEC2‐related Intellectual Disability Should Be Part of Rett Spectrum
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News Small Molecule Aids Motor Skills, Breathing in Rett Mouse Model
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News, syndicated COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say
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News, syndicated NORD’s Caregiver Respite Program Continues Through Pandemic
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News, syndicated Art Contest Salutes Creativity of Rare Disease Communities
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News Girls With Rett Have Higher Rate of Menstrual Seizures, Study Says
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News, syndicated NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum
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News Distinct Immune Protein Pattern Found in Rett Patients in Small Study
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News Anavex 2-73 Seen to Safely Ease Rett Severity in Women in Phase 2 Trial
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News Early Work Using Lab-grown ‘Mini-brains’ Identifies 2 Potential Rett Therapies
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News Anavex 2-73 Treatment for Rett to Receive New US Patent Broadening Coverage
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News, syndicated NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting
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News, syndicated Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
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News Reverse Rett Hosts Dec. 5 Virtual Big Night In Fundraiser, Benefitting Research
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News GW Stops ARCH Phase 3 Trial Testing Epidiolex in Children, Adolescents, Due to COVID-19
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News, syndicated Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
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News Adults With Rett Require Tailored Care Program to Manage Symptoms, Study Says
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News Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties
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News Taysha Gene Therapies, Catalent Partner to Advance Gene Therapies for Rett, Other Disorders
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News ‘Band of Mothers’ Connect to Raise Awareness About Boys With Rett
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News, syndicated NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
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News Rett Syndrome Research Trust Seeks Families for Global Registry
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News, syndicated UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
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News Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds
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News, syndicated Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
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News FDA Acts to Support Development of Potential Gene Therapy, TSHA-102
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News New KIF1A Gene Mutation Linked to Rett, Ends One Girl’s 15-year Diagnostic Journey
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News, syndicated Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
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News Nearly $500K Awarded to Support Search for Rett Syndrome Biomarkers
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News Events, Activities to Take Place Throughout October, Rett Syndrome Awareness Month
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News Overground or Treadmill Walking Suitable to Assess Gait in Rett Patients, Study Finds
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News, syndicated Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US
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News Brain Adenosine System May Be Therapeutic Target in Rett, Mouse Study Reports
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News Study Reveals Similar Spinal Surgery Outcomes in Rett Syndrome and Cerebral Palsy
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News Medical Advisory Board to Set Best-in-Class Care, Rettsyndrome.org Says
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News Rett Patients With Milder Mutation Have Poorest Quality of Life, Study Finds
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News First US-based Anavex 2-73 Trial Completed; Top-line Results Due by Year’s End
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News Vagus Nerve Stimulation Plus Sound May Improve Auditory Processing in Rett, Study Suggests
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News, syndicated Same But Different Photo Contest Celebrates People With Rare Diseases
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News Study Links Word Acquisition With Walking Ability in Rett Syndrome
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News AVXS-201, Gene Therapy for Rett, Again Moving Toward Clinical Trial, AveXis Says
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News, syndicated Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
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News Parents of Rett Children in Spain Struggle With Healthcare Access, Time Management, Study Reports
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News Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett
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News, syndicated EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
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News, syndicated New Streaming Channel Showcases Rare Disease Films
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News DNA Methylation Patterns Before Birth and After May Help in Understanding Rett
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News Taysha Raises $95M to Help Speed Clinical Development of Gene Therapies
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News Novel Function of MeCP2 Protein Could Be Route to Rett Therapy, Study Suggests
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News High Stress Levels Found in 40% of Parents Caring for Rett Child in Italy
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News RNA Editing Repairs Rett Syndrome Mutation in Mouse Model
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News BRD4 Protein May be Therapeutic Target for Rett Syndrome, Study Suggests
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News, syndicated National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
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News First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett
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News New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome
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News Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model
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News, syndicated Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
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News US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal
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News Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US
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News Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy
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News Coalition Will Address Racial Disparities in Rare Disease Communities
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News Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett
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News, syndicated COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
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News Blocking P2X7 Receptors May Be Therapeutic Target for Rett, Mouse Study Shows
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News, syndicated Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
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News Scientists Who Discovered MECP2 Link with Rett Syndrome Awarded 2020 Brain Prize
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cystic fibrosis, News, syndicated Video Games Connect Chronically Ill Children Isolated at Home, Hospital
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News, syndicated Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
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News Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders
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News, syndicated Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
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News Newron Terminates Sarizotan Development Due to STARS Trial Failure
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News, syndicated Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
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News Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model
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News, syndicated NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
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News Altered Nerve Cell Communication Tied to Motor Difficulties of Rett
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News Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment
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News NORD’s Advice on COVID-19: You’re Stronger Than You Think
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News Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits
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News NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
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News Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome
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Just Published, News Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
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News Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases
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News Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon
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News Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
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News MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds
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News FDA Grants Rare Pediatric Disease Designation to Trofinetide for Rett Syndrome
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News Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
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News Restoring Wnt6 Protein Eases Behavioral, Motor Impairment, Mouse Study Suggests
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