Curing Rett Syndrome: How Do We Get There
On April 23rd in New York City RSRT presented an event entitled “Curing Rett Syndrome – How Do We Get There?” The event was videotaped and is now available on RSRT’s YouTube channel.
Curing Rett Syndrome – How Do We Get There?
Ben Philpot, Ph.D.
Gene Awakenings for the Treatment of Neurological Disorders
Rett Syndrome Featured in GTC Conference on Rare Disease Drug Development and Commercialization
Rare disease has captured the interest of industry, and companies are looking to invest in orphan drug development. Monica Coenraads, RSRT Executive Director, joins leaders from big pharma, regulatory agencies, NIH, venture capitalists and academia to discuss key issues such as patient advocacy, pricing, and partnerships.
RSRT Highlighted in FasterCures Publication
RSRT joins Cystic Fibrosis Foundation, JDRF, Michael J. Fox Foundation, Leukemia & Lymphoma Society and others in a report that highlights insights and perspectives from leaders of 20 venture philanthropy groups that are transforming the cure enterprise.
RSRT Awards $4.2 million to Research
RSRT has made, for the second year in a row, the largest research commitment of any Rett non-profits worldwide.
Rett Researchers Get Up Close and Personal
In mid November of 2012 RSRT organized a three-day meeting of Rett scientists.
BioWorld Insight Highlights Rett Syndrome
A recent issue of BioWorld Insight, the weekly newsletter that provides behind-the-scenes analysis and commentary on the biotechnology industry, included a feature article on Rett Syndrome. The piece explored how recent interest in rare diseases on the part of pharmaceutical/biotech industry may impact research in disorders such as Rett Syndrome and Fragile X.
Prof. Bird Gives Speech at RSRT UK London Event
We invite you to listen to Prof. Bird’s remarks on the vitality of Rett research, the importance of maintaining global efforts in the field, and the possibilities that lie ahead.
RSRT Awards Funding Totaling $300,000 to Jeannie Lee
Jeannie Lee of Harvard is the newest recipient of RSRT funding. Her lab will join the ongoing effort to reactivate the silent MECP2 gene. Visit our blog to learn more.
RSRT Awards Continued Funding to Zoghbi Lab Bringing Total Commitment to $817,0000
Additional $1/2 million was awarded to Huda Zoghbi, M.D. to explore three novel approaches to potentially treating Rett Syndrome. Visit our blog to learn more.
RSRT Awards Continued Funding to Kipnis Lab Bringing Total Commitment to $627,0000
The Trustees of RSRT recently awarded $440,000 of continuation funding to Jonathan Kipnis, Ph.D. of the University of Virginia to explore immune function in Rett Syndrome.
RSRT Awards Continued Funding to Modifier Screen Project Bringing Total Commitment to $800,0000
The Trustees of RSRT recently awarded $300,000 of continuation funding to Monica Justice, Ph.D. of Baylor College of Medicine for her ongoing MECP2 modifier screen project. We believe this project is one of the most intriguing and potentially clinically relevant projects in the Rett research arena.
RSRT Scientific Advisory Board member, Huda Zoghbi, Wins Prestigious Gruber Neuroscience Prize
Huda Y. Zoghbi, MD, will receive the 2011 Neuroscience Prize of The Peter and Patricia Gruber Foundation for her pioneering work in unlocking the genetic and molecular mysteries behind a number of devastating neurological disorders, including Rett syndrome, spinocerebellar ataxia type 1, and brain tumors called medulloblastomas. Her contributions to these discoveries have greatly advanced our scientific understanding not only of these disorders, but also of more common ones, including autism, Parkinson’s disease, and Alzheimer’s disease. She will receive the award November 13 in Washington D.C. at the Annual Meeting of the Society for Neuroscience and will deliver a lecture titled “Rett syndrome: Linking Epigenetics and Neuronal Plasticity.”
2010 Audited Financials Online
RSRT is lean and once again our financials prove it – in 2010 96% of every dollar raised was channeled directly to our research program. In the spirit of complete transparency and accountability our audited financial statements and 990 are available on our website. We take this opportunity to thank our supporters. We are able to pursue our important work because of you.
RSRT Funds New Project in the lab of Mark Bear, MIT
RSRT is pleased to welcome Dr. Mark Bear of MIT as a recipient of research support. Dr. Bear is a pioneering researcher whose thoughtful insight has dramatically advanced the understanding of Fragile X. His seminal work was featured in Forbes late last year.
Dr. Bear has proposed that just as Fragile X is due to over-synthesis of proteins at the synapse, Rett may be due to under-expression of protein at the same locations. RSRT has committed funding to the Bear lab to first test this hypothesis and if proven correct to explore whether pharmacological manipulations of mGluR signaling will improve any of the mutant mice Rett-like symptoms. The in vivo mouse work will be performed in collaboration with Professor Adrian Bird.
The RSRT Blog recently featured an interview with Dr. Bear.
RSRT Trustee, Adrian Bird, Wins Prestigious Gairdner International Award
On March 23rd Gairdner Foundation of Canada announced the winners of its prestigious International Awards. RSRT Trustee, Professor Adrian Bird, was awarded the prize for his discoveries in the field of DNA methylation and its role in gene expression. The awards are one of the most prestigious medical honors in the world and are renowned for being early predictors of Nobel Prize winners. In the past decade, Gairdner winners have won 19 of the 26 Nobel Prizes in physiology or medicine.
Read The Lancet article.
RSRT Advisor, Huda Zoghbi, Takes the Helm of World’s First Pediatric Neurological Research Institute
The recently opened Jan and Dan Duncan Neurological Research Institute (NRI) in Houston, Texas is dedicated to scientific exploration of childhood neurological disorders. Director and RSRT Scientific Advisory Huda Zoghbi, whose laboratory established that mutations in MECP2 cause Rett Syndrome, envisioned a center where researchers with diverse interests could work within an environment of ongoing, cross-disciplinary dialogue. The soaring new structure is located in the heart of the Texas Medical Center, close to the basic science campus of Baylor College of Medicine and Texas Children’s Hospital.
Read a recent interview with Dr. Zoghbi on the RSRT Blog.
RSRT on The View
Rett Syndrome received national TV media attention on January 28th when the co-hosts of The View interviewed Manny and Stefanie Gutierrez about their daughter Anna and their journey with Rett Syndrome, and Monica Coenraads, Executive Director of RSRT.
May 2010 Issue of Drug Discovery News Features RSRT