Updates

October 30, 2020October 30, 2020
Rett Syndrome Research Trust Seeks Families for Global Registry

As the Rett Syndrome Research Trust (RSRT) prepares to launch ... Read more
October 28, 2020October 28, 2020
UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

A photo of a bespectacled young boy, his red baseball ... Read more
October 23, 2020October 23, 2020
Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds

Treatment with the antidepressant mirtazapine preserves motor and respiratory function, ... Read more
October 21, 2020October 21, 2020
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the ... Read more
October 16, 2020October 16, 2020
FDA Acts to Support Development of Potential Gene Therapy, TSHA-102

The U.S. Food and Drug Administration (FDA) has granted orphan drug and ... Read more

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Updates

Rett Syndrome Research Trust Seeks Families for Global Registry

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

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

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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

New Streaming Channel Showcases Rare Disease Films

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

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

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

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

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

RNA Editing Repairs Rett Syndrome Mutation in Mouse Model

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

Rett Syndrome Research Trust Seeks Families for Global Registry

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

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

Subscribe to our mailing list

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

New Streaming Channel Showcases Rare Disease Films

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

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

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

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

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

RNA Editing Repairs Rett Syndrome Mutation in Mouse Model

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

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