These “awakenings” lasted a few days to a week and have…, Dr. Daniel Tarquinio, the director of the Rett Syndrome Clinic in Atlanta will soon be recruiting for a 10 person, open label trial (everyone gets the drug) testing triheptanoin. âFrom a safety perspective, this includes planning for disease-specific challenges to patient accrual and retention to maximize the size of the premarket safety dataset. Can you introduce yourself and tell us a little about your career? Rett Syndrome Awareness Month is here! Great achievements happen when people stand up and say I’m in. RSRT received an influx of emails and phone calls asking for more information. To accelerate full spectrum research to cure Rett syndrome and empower families with information, knowledge and connectivity. Although we personally are…, I’m proud to say that RSRT once again received a three-star rating from Charity Navigator, the organization that assesses the transparency, financial health, and efficiency of non-profits. Thank you to all our supporters!
Much love, Kathy Hunter, Your email address will not be published. Her scoliosis is turning her into a question mark, her back brace is so uncomfortably tight…, As always at RSRT, our funded projects are aimed at developing effective treatments and a cure for Rett Syndrome. More than anything. Despite its debilitating nature, Rett offers three silver linings: Together, these three facts scream out for a genetic solution to Rett. Breakthrough discoveries are made in academic labs. We invite you to view our infographic. We are struck by the following quote from Dr. Green:…, The Milken Institute Global Conference, which explores solutions to pressing challenges including healthcare, took place today in Los Angeles. Before starting at RSRT I had met two girls with Rett Syndrome—my own daughter and…, There is no mystery about why a girl suffers from Rett Syndrome. His list of contributions to the Rett field are numerous starting with the discovery of the MeCP2 protein…. DBS is a surgical procedure that involves implanting electrodes in specific areas of…, Later this month, from the 17th to the 21st, about 30,000 neuroscientists will gather in Chicago for the annual meeting of the Society for Neuroscience.
It’s also what predisposes or protects us from disease. On this occasion, the consortium…. Among these are two RSRT-funded papers: the gene therapy paper from Gail Mandel and Adrian Bird and the statin paper from Monica Justice. It’s…, Dear Friends, I am sitting in a plane on the tarmac at Chicago O’Hare, returning home from an amazing event that was spearheaded by a Rett family.
Dangerous precedent for the FDA? Rather than target the gene or the RNA, this strategy delivers the healthy MECP2 protein on an ongoing basis. It doesn’t cost you anything extra and is an easy way to give back. ©2020 Girl Power 2 Cure, Inc. All Rights Reserved. There are also revised updates to a section on natural history studies, including a discussion about how such studies might be used to measure a proposed therapy’s benefits, and the need for disease biomarkers. Her arms had stopped moving and she was breathing quietly. RSRT has fared very well for several reasons—our low overhead (an average of 4% of our expenses are spent on administration…, It started out as a pretty good summer day. Multiple mutations on MeCP2 In most cases of Rett syndrome, one mutation occurs on (a single copy of) the MeCP2 gene. The look that says, “I understand you.“ A close friend and single mother once said to me how important it was to have someone bear witness to her daily life caring for her…, In August of 2014, at the age of two, our son Bo was diagnosed with MECP2 Duplication Syndrome. It goes without saying that Monica’s knowledge, passion and courage provide not only the backbone but the spirit of…, Three years ago, a study showed that a bone marrow transplant performed in pre-symptomatic male mice models of Rett Syndrome substantially extended their lives and reduced symptoms of disease. Brad has been a strong and steadfast advocate and fundraiser for Rett research and RSRT. Founded on scientific knowledge generated over many years, in early 2017 RSRT launched a three-year, $33 million research plan, Roadmap to a…, Dear families and supporters, We are excited to share the statement below from AveXis/Novartis regarding their AVXS-201 gene therapy program for Rett Syndrome. While a positive MECP2 genetic test confirms the diagnosis, it is not required. It seems like almost every day, breakthrough scientific discoveries provide novel insights that could result in new and better ways to treat your children. A…, I have a soft spot for Rett grandparents. This year in our theology classes we were given a project where we had to choose an important topic to us and make a difference in the world. We simply must raise the full amount. It refers to changes in gene expression caused…, Glia in Rett Syndrome: New Findings Gail Mandel’s new work reveals not only the important information that MECP2 is expressed in glia as well as in neurons, but the discovery that MECP2-deficient astrocytes (a subset of glia) seem to effectively stunt the development of neurons.
Every time I have a conversation with these fellow parents I learn something new and am inspired by their strength and by…, I’ll let you in on a secret. Our kids are often tortured by constipation, reflux, gas pains and more.
Our mission has always been a cure, not an improvement of some degree in a symptom or two… an all-out cure. 3 W Garden St We invite you to be part of the solution. He discovered the Rett gene, MECP2, and he made the first animal model of the disease. AVXS-201 Gene Therapy Phase 1 (RSRT/Novartis-AveXis, anticipated), https://reverserett.org/avexis-reports-rett-gene-therapy-program-avxs-201/, Keep up the good work! As you know, the Consortium is charged with developing gene therapy techniques that could treat or significantly reverse the symptoms of Rett.
The drug is already in clinical trials for Cystic Fibrosis and Duchenne’s muscular dystrophy. Since no two individuals with Rett syndrome are the same, we need multiple treatment approaches.
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