September 18, 2020September 18, 2020

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

Rettsyndrome.org has launched the International Rett Syndrome Foundation Medical Advisory ... Read more

Updates

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

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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

First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett

New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome

Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal

Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US

Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy

Coalition Will Address Racial Disparities in Rare Disease Communities

Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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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

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

Subscribe to our mailing list

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

First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett

New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome

Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal

Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US

Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy

Coalition Will Address Racial Disparities in Rare Disease Communities

Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Blocking P2X7 Receptors May Be Therapeutic Target for Rett, Mouse Study Shows

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