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The Rett Syndrome Research Trust (RSRT) is the patient advocacy

https://reverserett.org/
As the largest funder of Rett syndrome research worldwide, RSRT has played a vital role in initiating and evolving the trajectory of progress toward cures. All genetic medicines in development by biopharma have leveraged discoveries and resources made possible by RSRT.

Cures | Rett Syndrome Research Trust

https://reverserett.org/cures/
Where We've Been. Built on decades of scientific advances, Roadmap to Cures will select and drive innovation for three genetic medicines that attack the root cause of Rett syndrome, and advance into clinical trials by 2028. Key Information. Take Action. Genetic medicines are made up of two parts: the cargo. which corrects for the mutation, and the.

RSRT launches program to advance genetic therapies for Rett

https://rettsyndromenews.com/news/rsrt-launches-program-advance-genetic-therapies-rett/
by Andrea Lobo, PhD June 12, 2024. Rett Syndrome Research Trust (RSRT) has launched an initiative to advance three genetic medicines for Rett syndrome to clinical trials by 2028. Called Roadmap to Cures, it seeks to raise $40 million over the next four years to help select and develop the therapies and is based on the new MECP2 Editing

Developing A NextGen RNA Therapy for Rett Syndrome - YouTube

https://www.youtube.com/watch?v=e4AkoP5kcGs
David Huss, PhD, VP, Head of Research for Shape Therapeutics discusses the company's proprietary RNA technologies and how they will be applied to fix MECP2 m

Rett Syndrome Research Trust Launches Roadmap to Cures to - GlobeNewswire

https://www.globenewswire.com/news-release/2024/06/05/2893886/0/en/Rett-Syndrome-Research-Trust-Launches-Roadmap-to-Cures-to-Advance-Next-Generation-Genetic-Medicines-into-Clinical-Trials.html
About The Rett Syndrome Research Trust. RSRT is the patient advocacy organization working to cure Rett syndrome. As the largest funder of Rett syndrome research worldwide, RSRT has played a vital

About the Rett Syndrome Research Trust

https://reverserett.org/about-rsrt/
Brad serves on the Board of Rett Syndrome Research Trust (RSRT) and in 2015 founded Reverse Rett Chicago, a fundraiser that has generated over $3 million for Rett syndrome research since inception. Brad also serves on the Board of Aspiritech, a non-profit that employs adults with autism. Brad previously served as Treasurer of the Illinois

Rett Syndrome Research Trust - YouTube

https://www.youtube.com/user/reverserett
The Rett Syndrome Research Trust is singularly focused on a cure for Rett syndrome and related MECP2 disorders. Achieving success takes a deep understanding of the relevant science, an environment

Trofinetide for the treatment of Rett syndrome: a randomized ... - Nature

https://www.nature.com/articles/s41591-023-02398-1
J.L.N. has received research funding from the International Rett Syndrome Foundation, the National Institutes of Health and Rett Syndrome Research Trust; and personal consultancy fees from Acadia

Our Research - International Rett Syndrome Foundation

https://www.rettsyndrome.org/research/our-research/
Our strategy. IRSF's Scientific Strategy consists of three guiding pillars that cover Rett research at every level: Critical research - Critical research addresses gaps in researchers' knowledge of Rett syndrome, including MECP2 and other targets that give rise to the pathology of Rett. It equips researchers with the information they need

Consensus guidelines on managing Rett syndrome across the lifespan

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7488790/
Patient family groups (International Rett Syndrome Foundation and Rett Syndrome Research Trust), represented by parents of individuals with RTT (PN and MC), were involved in the development of the patient care guidance and writing of this manuscript. Their organisations will assist with dissemination of the guidance.

Rett Syndrome Research Trust - National Organization for Rare Disorders

https://rarediseases.org/organizations/rett-syndrome-research-trust/
The Rett Syndrome Research Trust (RSRT) is the organization working to cure Rett syndrome. As the largest funder of Rett syndrome research worldwide, RSRT has played a vital role in initiating and evolving the trajectory of progress toward a cure. All genetic therapies in development by biopharmaceutical companies have leveraged discoveries and

Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage

https://ncats.nih.gov/news-events/news/rett-syndrome-research-trusts-genetic-medicines-summit-sets-stage-accelerated
Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development. Last updated on October 31, 2023. 6701 Democracy Boulevard Bethesda, MD 20892-4874 301-594-8966 ncatsinfo@nih.gov. Connect with us. For language access assistance, contact the NCATS Public Information Officer.

Advanced genetic therapies for the treatment of Rett syndrome: state of

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10248472/
Introduction. Rett syndrome (RTT; OMIM 312750) is an X-linked neurodevelopmental disorder that almost exclusively affects girls (Amir et al., 1999).With an incidence of 1 in 10,000 it represents the most common genetic cause of severe intellectual disabilities in females worldwide (Neul et al., 2010).Apparently normal at birth, typical (or classic) RTT patients seem to grow up fine through the

Where We've Been | Rett Syndrome Research Trust

https://reverserett.org/cures/progress/
In 2007, Professor Bird shocked the scientific and Rett communities by showing that Rett symptoms in mice models disappeared upon restoration of MECP2. This finding changed everything as it suggested a cure was possible. Monica Coenraads launched the Rett Syndrome Research Trust in 2008 to focus exclusively on cures.

UMass Chan scientists receive $2.3 million from Rett Syndrome Research

https://www.umassmed.edu/news/news-archives/2024/03/umass-chan-scientists-receive-$2.3-million-from-rett-syndrome-research-trust-for-new-research
The funding received by UMass Chan is part of $10.3 million in new grants awarded by the Rett Syndrome Research Trust in 2023. "I'm so pleased to be able to put RSRT's resources behind Erik, Jonathan, and Scot, leaders in their field who are advancing cutting edge editing technologies and applying them to Rett syndrome," said Monica

About Rett Syndrome | Neurogene

https://www.neurogene.com/patients-and-families/about-rett-syndrome/
The Rett Syndrome Research Trust has a singular mission: a cure for Rett syndrome. Our remarkable progress is made possible by the support, passion, and commitment of Rett families around the world. Learn More >> International Rett Syndrome Foundation.

Rett Syndrome Research Trust

https://www.coriell.org/1/Rett-Syndrome
The Rett Syndrome Research Trust (RSRT) is a non-profit organization dedicated to curing Rett syndrome and is offering a new collection of induced pluripotent stem cells (iPSCs) for use in research. RSRT funded a study that collected blood and tissue from individuals with Rett syndrome in order to generate a stem cell resource that facilitates

Rett Syndrome Research Trust launches parent-reported registry

https://rettsyndromenews.com/news/parent-reported-registry-launched-rett-syndrome-research-trust/
The Rett Syndrome Research Trust (RSRT) has launched a global registry for parents to share their knowledge and experiences about caring for their loved ones with Rett syndrome. Called the Rett Syndrome Global Registry , its aim is to advance the development of genetic medicines for Rett.

Rett Syndrome Research Trust Partners with Vivalink to Shift Symptom

https://www.prnewswire.com/news-releases/rett-syndrome-research-trust-partners-with-vivalink-to-shift-symptom-assessment-paradigms-in-rett-syndrome-302124342.html
About Rett Syndrome Research Trust RSRT is the patient advocacy organization working to cure Rett syndrome. As the largest funder of Rett syndrome research worldwide,

Consensus guidelines on managing Rett syndrome across the lifespan

https://www.rettsyndrome.org/wp-content/uploads/BMJ-Consensus-guidelines-Sept-2020.pdf
Foundation and Rett Syndrome Research Trust), repre-sented by parents of individuals with RTT (PN and MC), were involved in the development of the patient care guidance and writing of this manuscript. Their organisa-tions will assist with dissemination of the guidance. RESULTS The guidance was formulated into a checklist ( 1) table

Homepage - International Rett Syndrome Foundation

https://www.rettsyndrome.org/
Rett syndrome is a rare genetic neurological disorder that leads to severe impairments, affecting nearly every aspect of life. Rett particularly affects speech, purposeful hand use, and coordination, leaving individuals understanding more than they can communicate. Caused by a gene mutation, Rett is first recognized in infancy and occurs

About Rett Syndrome, Symptoms, Causes, Diagnosis and Treatments

https://reverserett.org/about-rett-syndrome/
Although there currently is no cure for people with Rett syndrome, the sole focus of the Rett Syndrome Research Trust is to deliver cures as quickly as possible. We are optimistic because Rett-like symptoms are dramatically reversible in animal models of the disorder. Curing the disorder requires targeting the root cause: mutations in the MECP2

Rett Syndrome - National Institute of Neurological Disorders and Stroke

https://www.ninds.nih.gov/health-information/disorders/rett-syndrome
Rett syndrome is a neurodevelopmental disorder. It is characterized by typical early growth and development, which is then followed by: Other symptoms may include sleep problems, teeth grinding, and difficulty chewing. Age of onset, severity of symptoms, and how Rett syndrome progresses varies from child to child.