RSRT (@rsrt) 's Twitter Profile
RSRT

@rsrt

The Rett Syndrome Research Trust: Our singular focus is a cure for Rett syndrome.

DONATE: reverserett.org/donate/

ID: 25153715

linkhttp://www.reverserett.org calendar_today18-03-2009 21:01:36

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Learn more about #genetherapy, the value to #patients & policy updates needed to #EnsureAccess. Register for Institute for Gene Therapies event "Realizing the Promise of Gene Therapy: Ensuring Access" on 6/26 to hear from patient advocates and IGT leaders. forms.gle/u6hFmagnCeaLQs…

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Joyfully Josie is a heartwarming new book aimed at introducing disabilities, medical complexities, and inclusion to young children. The book is about Josie, who has FOXG1 syndrome, a neurological disorder similar to #Rettsyndrome. Check it out! joyfullyjosie.love

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Don’t forget – tune in to our virtual R&D Day today at 10 a.m. ET to hear the latest in the development of our investigational #genetherapies in giant axonal neuropathy (GAN) and Rett syndrome. bit.ly/3NPTiM3

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Reverse Rett Colorado is coming up on July 28. Thank you to grandparents Karen and Steve Bye for spearheading this beautiful event. Way to make a difference to the research! reverserett.org/events/reverse…

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FDA has just approved a gene therapy for hemophilia A. BioMarin’s president of R&D, Henry Fuchs, sums it up as follows: “The learning here was it doesn’t work in everybody. It doesn’t last forever. Our view is it works in enough people and long enough.” endpts.com/biomarins-hemo…

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An exciting initiative funded by NIH uses the latest genome editing tools to advance cures for #Rettsyndrome and 3 other diseases! The lead on the effort is Dr. Cathleen Lutz of The Jackson Laboratory Learn more on our blog. reverserett.org/news/articles/…

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RSRT is laser-focused on genetic medicines. Gene therapy is a genetic medicine that delivers healthy genes to compensate for mutated ones. Learn more about this approach with this short explainer from the American Society of Gene and Cell Therapy. patienteducation.asgct.org/gene-therapy-1…

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With Emerald, doing nothing is changing everything. Dina Kitabi explains how her sci fi tech of the future is revolutionizing Parkinson’s. With RSRT funding we’re excited about what Emerald can do for Rett. youtube.com/watch?v=TLxiiJ…

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Neurogene's CEO, Dr. Rachel McMinn, provides details on their Rett gene therapy trial. A short registration form is required to access the webcast. edge.media-server.com/mmc/p/q3vx354g…

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See Rett syndrome through Emerald eyes! Our latest blog by our CEO, Monica Coenraads, shows how Emerald quantifies symptoms in ways humans simply can’t. reverserett.org/news/articles/…

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Taysha announces that they are cleared to dose Patient #2. Update on Patient #1 will be shared mid-August. reverserett.org/news/articles/…

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Our CEO Monica Coenraads was recently interviewed on a Turn Autism Around Podcast. Watch it for a thorough summary of what causes Rett and the current state of the research. marybarbera.com/rett-syndrome-…

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We've awarded additional funding to Pete Beal to advance an RNA editing project. This genetic-based approach aims to correct the mutations that cause Rett by harnessing a natural protein already in cells. reverserett.org/news/articles/…

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Taysha has shared a community letter regarding their clinical trials in Canada, US and UK. reverserett.org/news/articles/…

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Help advance a cure for Rett by participating in the Rett Syndrome Global Registry. rettglobalregistryx.acrossmatrix.com/en-US/#/user-r…