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May 15, 2020May 15, 2020

Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders

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Taysha Gene Therapies has raised $30 million and established a partnership with UT Southwestern Medical ... Read more

May 12, 2020May 12, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

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The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand ... Read more

May 8, 2020May 8, 2020

Newron Terminates Sarizotan Development Due to STARS Trial Failure

News

Treatment with sarizotan failed to reduce apnea episodes (breathing stops) while awake compared to a ... Read more

April 28, 2020April 28, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

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While there are few silver linings to the cloud created by COVID-19, the pandemic that ... Read more

April 24, 2020April 24, 2020

Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model

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A form of gene therapy based on providing an unstable version of the MECP2 gene ... Read more

April 22, 2020April 24, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

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The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people ... Read more

April 17, 2020April 17, 2020

Altered Nerve Cell Communication Tied to Motor Difficulties of Rett

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Problems with nerve cell communication and adaptation in the brain’s motor cortex is associated with ... Read more

April 10, 2020April 10, 2020

Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment

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April 6, 2020April 6, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

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April 3, 2020April 3, 2020

Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits

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Recent Posts

Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders

May 15, 2020
Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

May 12, 2020
Newron Terminates Sarizotan Development Due to STARS Trial Failure

May 8, 2020

Featured Posts

Parents of Rett Patients View Deep Brain Stimulation as Viable Option

December 17, 2019

DNA Sequencing Identifies Unusual Genetic Cause of Rett-like Disease in Young Girl

January 17, 2020

FDA, Newron Pharma to Meet in Step to Releasing Results of Sarizotan Pivotal Trial

December 13, 2019

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