@RSRT

The Rett Syndrome Research Trust: Our singular focus is a cure for Rett syndrome. DONATE: https://nitter.cf/t.co/R06XhvrgBw

 Connecticut
reverserett.org
Joined March 2009
  • Tweets1.6K
  • Following299
  • Followers2K
418Media
Help advance a cure for Rett by participating in the Rett Syndrome Global Registry. rettglobalregistryx.acrossma…
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Taysha has shared a community letter regarding their clinical trials in Canada, US and UK. reverserett.org/news/article…

Taysha Gene Therapies doses patient #2 and provides an update on their clinical trial plans in...

Taysha Gene Therapies doses second patient in gene therapy clinical trial and provides an update on their clinical trial plans in Canada, US and UK.

reverserett.org
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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/article…

RSRT Doubles Down on RNA Editing for Rett Syndrome with Additional Funding to Beal Lab at UC Davis

The Rett Syndrome Research Trust (RSRT) is funding several cutting-edge approaches that target the genetic root cause of Rett syndrome. One of the most promising is RNA editing: changing RNA to...

reverserett.org
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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-syndrom…

The Power of Gene Therapy: Unlocking Hope for Rett Syndrome and ASD - Dr. Mary Barbera

Dr. Mary Barbera and Monica Coenraads explore the potential of gene therapy to transform the lives of individuals with Rett Syndrome and Autism Spectrum Disorder (ASD).

marybarbera.com
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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/article…

Taysha Cleared to Dose Second Patient in Gene Therapy Trial

Taysha Cleared to Dose Second Patient in Gene Therapy Trial

reverserett.org
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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/article…

Understanding Rett through the Power of Artificial Intelligence

Earlier this year RSRT announced a $1.1 million award to Dina Katabi and her team at Emerald Innovations to expand their work using the Emerald device to assess symptoms of irregular breathing, sleep...

reverserett.org
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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/…
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Congratulations to everyone at @NeurogeneInc and @neoleukin
Neurogene Inc.@NeurogeneInc
18 Jul 2023
We are excited to announce that we have entered into a merger agreement with @Neoleukin to create a Nasdaq-listed biotech company focused on advancing our differentiated portfolio of genetic medicines for complex neurological diseases. Learn more: bit.ly/3DgReqi
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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=TLxiiJ8S…

Using Invisible Radio Waves To Detect Parkinson’s With Help From AI

Dina Katabi is a prominent computer scientist known for her groundb...

youtube.com
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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/g…

What is Gene Therapy?

Gene therapy is the use of genetic material to treat or prevent disease. Learn more about the basics of gene therapy, the potential for this growing field…

patienteducation.asgct.org
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We aren't the only species that edits our RNA. Check out what octopuses can do! npr.org/2023/06/08/118100921…

Octopuses tweak the RNA in their brains to adjust to warmer and cooler waters

The California two-spot octopus can edit the RNA in its brain to produce different proteins as ocean temperatures fluctuate, a new study finds.

npr.org
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Happy 4th of July to everyone celebrating!
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CRISPR is not the only genome editing tool in town -- meet "Fanzor!" news.mit.edu/2023/fanzor-sys…

Researchers uncover a new CRISPR-like system in animals that can edit the human genome

Researchers have discovered the first programmable RNA-guided system discovered in eukaryotes. The technology, known as Fanzor, has the potential to be more easily delivered to cells and tissues than...

news.mit.edu
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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 @jacksonlab Learn more on our blog. reverserett.org/news/article…

Lutz and Liu Dream Team Awarded $23 Million to Cure Four Rare Diseases: Rett is One of Them

Just 11 years ago, the development of CRISPR genome editing launched a new era in biology and medicine, with new possibilities to treat genetic diseases. Now, an ambitious new initiative aims to use...

reverserett.org
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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-hemophi…

Updated: BioMarin’s hemophilia A gene therapy Roctavian wins FDA approval after delay and rejecti...

The FDA approved the first gene therapy to treat the genetic bleeding disorder hemophilia A on Thursday, capping a turbulent regulatory saga for BioMarin, which will ...

endpoints.news
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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/rever…

Reverse Rett Colorado 2023 - Rett Syndrome Research Trust

Rett Syndrome Research Trust advances medical research, news and information about Rett Syndrome.

reverserett.org
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Brief update from @TayshaGTx on Patient #1 in their Rett gene therapy trial. ir.tayshagtx.com/news-releas…

Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant...

Company views that results of comprehensive data analysis of TSHA-120 and development of disease progression model (DPM) address U.S. Food and Drug Administration (FDA) feedback regarding the...

ir.tayshagtx.com
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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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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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Learn more about #genetherapy, the value to #patients & policy updates needed to #EnsureAccess. Register 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/u6hFmagnCeaLQspLA

Realizing the Promise of Gene Therapy: Ensuring Access

The Institute for Gene Therapies will host a reception for Congressional staff, patient advocates, scientific and academic stakeholders, and leaders in the gene therapy space to provide an overview...

docs.google.com
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