Updates

November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to ... Read more
November 25, 2020November 25, 2020
Reverse Rett Hosts Dec. 5 Virtual Big Night In Fundraiser, Benefitting Research

Viewers from around the world are invited to join in ... Read more
November 20, 2020November 20, 2020
GW Stops ARCH Phase 3 Trial Testing Epidiolex in Children, Adolescents, Due to COVID-19

Due to the unprecedented challenges caused by the ongoing COVID-19 ... Read more
November 19, 2020November 19, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Leaders in the U.S. rare disease community came together recently ... Read more
November 18, 2020November 18, 2020
Adults With Rett Require Tailored Care Program to Manage Symptoms, Study Says

The transition into adulthood of women with Rett syndrome warrants ... Read more

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Updates

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

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

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

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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

Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties

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

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

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

Rett Syndrome Research Trust Seeks Families for Global Registry

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Same But Different Photo Contest Celebrates People With Rare Diseases

Study Links Word Acquisition With Walking Ability in Rett Syndrome

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

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

Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

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

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

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

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

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

Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties

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

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

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

Rett Syndrome Research Trust Seeks Families for Global Registry

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

Subscribe to our mailing list

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Same But Different Photo Contest Celebrates People With Rare Diseases

Study Links Word Acquisition With Walking Ability in Rett Syndrome

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

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

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

Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US