Rett Syndrome Current Events

Rett Syndrome Current Events, Rett Syndrome News Articles.
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Rett syndrome research reveals high fracture risk
Researchers at Perth's Telethon Institute for Child Health Research have found that girls and young women with Rett syndrome are nearly four times more likely to suffer a fracture. (2008-03-06)

7th World Congress for Rett Syndrome to convene in New Orleans, La., June 22-26
The 7th World Congress for Rett Syndrome will consist of four interconnected meetings targeting researchers, clinicians, medical professionals and families impacted by Rett syndrome. These meetings include a Family Education Conference and a Science Symposium. (2012-03-05)

Rettsyndrome.org supports further clinical trials with Trofinetide
Rettsyndrome.org today announced that the Board of Directors has made a financial commitment, through the Research to Reality Campaign, to continue support for further clinical trials with trofinetide, a drug developed and manufactured by Neuren Pharmaceuticals. (2015-10-13)

Genetic analysis reveals range of Rett syndrome
The first comprehensive analysis of the clinical effects of genetic mutations involved in Rett syndrome will enable affected families to receive a more accurate indication of their child's prognosis. (2008-03-11)

Study offers treatment hope for Rett syndrome
The symptoms of a severe autism spectrum disorder affecting at least 10,000 children in the UK could be reversed following research by Scottish scientists. (2007-02-08)

Yale researchers find potential treatment for Rett Syndrome
An experimental cancer drug can extend the life of mice with Rett Syndrome, a devastating genetic disorder that afflicts about one of every 10,000 to 15,000 girls within 6 to 18 months after birth, Yale researchers report June 10 in the journal Molecular Cell. (2020-06-10)

New insight into Rett syndrome severity
A research collaboration between Australia and Israel has identified a genetic variation that influences the severity of symptoms in Rett syndrome. (2009-04-20)

A new drug shows preclinical efficacy in Rett syndrome
A new article published in the Cell Reports describes how a new drug is able to reduce the symptoms and activate the dormant neurons characteristic of Rett Syndrome in preclinical models. (2018-05-14)

Rettsyndrome.org reaches milestone of $40 million in research funding
The International Rett Syndrome Foundation, now doing business as Rettsyndrome.org, announces today that Rettsyndrome.org has reached a milestone in research funding. The leading foundation for Rett research has distributed over $40 million dollars to accelerate treatments for Rett syndrome. (2017-01-09)

Effective drugs for Parkinson's reduce symptoms of Rett syndrome in mice
IDIBELL researchers, led by the director of the Program for Epigenetics and Cancer Biology, ICREA researcher and Professor of Genetics at the University of Barcelona, Manel Esteller, have shown that a combination of effective drugs for Parkinson's disease in mice that are used as a model of human Rett syndrome reduces some of the symptoms associated with this disease. (2014-06-16)

Insight into cause of brain disorders may aid quest for treatments
Fresh discoveries about a range of neurological disorders may inform the development of new therapies. (2017-03-27)

Melior enters drug discovery collaboration with Rett Syndrome Research Trust
Melior Discovery Inc. announced today that it has entered into collaboration with the Rett Syndrome Research Trust to screen drug candidates in an in vivo model of Rett syndrome. (2010-03-30)

Mental and physical exercise improves genetic mental impairment
Australian scientists have shown that mental and physical exercise can improve coordination and movement problems in Rett syndrome, a devastating genetic brain development disorder that primarily affects females. (2008-06-20)

Critical mass in rare diseases -- an innovative Internet approach
The internet is emerging as a valuable tool for scientists to gather data for critical research into rare diseases. (2008-10-02)

Neuren's Phase 2 trofinetide trial shows significant benefit in pediatric Rett syndrome
The International Rett Syndrome Foundation, now doing business as Rettsyndrome.org, announces today that Neuren Pharmaceuticals reported that trofinetide had significant clinical benefit in their Phase 2 clinical trial in girls with Rett syndrome aged 5 to 15. Rettsyndrome.org (a sponsor of this trial) is enthusiastic about the results and proud to partner with Neuren to move trofinetide forward toward the Phase 3 clinical trial. (2017-03-21)

Mecasermin (rh-IGF-1) treatment for Rett Syndrome is safe and well-tolerated
The results from Boston Children's Hospital's Phase 1 human clinical trial in Rett syndrome came out today. A team of investigators successfully completed a Phase 1 clinical trial using mecasermin (recombinant human insulin-like growth factor 1 [IGF-1]), showing proof-of-principle that treatments like IGF-1 which are based on the neurobiology of Rett syndrome, are possible. (2014-03-10)

'Dark genome' is involved in Rett Syndrome
Researchers at the Epigenetics and Cancer Biology Program at IDIBELL led by Manel Esteller, ICREA researcher and professor of genetics at the University of Barcelona, have described alterations in noncoding long chain RNA sequences in Rett syndrome. (2013-05-02)

Life threatening breathing disorder of Rett syndrome prevented
A group of researchers at the University of Bristol have sequestered the potentially fatal breath holding episodes associated with the autistic-spectrum disorder Rett syndrome. (2010-10-04)

Scientists create first animal model of Rett Syndrome
Researchers from the Whitehead Institute have created the long-awaited animal model for Rett syndrome, one of the most common causes of mental retardation in females. The transgenic mouse model sheds light on the underlying mechanism of the disease and suggests a new reason for hope in the research toward therapies. (2001-03-01)

New drug offers hope for devastating childhood disease
A powerful new drug which could relieve the symptoms of devastating childhood disease Rett syndrome is on the horizon thanks to a funding injection of £180,000. (2014-04-09)

Rettsyndrome.org names 14 Clinics as Clinical Research Centers of Excellence
Rettsyndrome.org, is launching an innovative clinic program, designating 14 clinics in the United States as Rett Syndrome Clinical Research Centers of Excellence. (2016-10-25)

Discovery of a new drug target could lead to novel treatment for severe autism
A novel drug target has been used to rescue functional deficits in human nerve cells derived from patients with Rett syndrome, a severe form of autism-spectrum disorder. The research could lead to a new treatment for Rett syndrome and other forms of autism-spectrum disorders. (2016-01-04)

Study points to window of opportunity for successful autism therapy
New evidence from studies of neurons derived from patients with a genetic condition known as Rett Syndrome suggests that there may be a critical period in development during which neuronal abnormalities could be corrected by drug treatments. That's the conclusion of researchers who report their findings in the Nov. 12 issue of Cell, a Cell Press publication. (2010-11-11)

New drug target for Rett syndrome
Harvard Stem Cell Institute researchers have identified a faulty signaling pathway that, when corrected, in mice ameliorates the symptoms of Rett syndrome, a devastating neurological condition. The findings could lead to the discovery of compounds or drugs that may benefit children affected by the disease, says neurobiologist Jeffrey Macklis, Max and Anne Wien Professor of Life Sciences in the Department of Stem Cell and Regenerative Biology, and Center for Brain Science, at Harvard University, who directed the work. (2016-02-02)

Rett Syndrome Association of Massachusetts and Rettsyndrome.org continue their partnership
The Rett Syndrome Association of Massachusetts and Rettsyndrome.org announce today their renewed commitment to work together to defeat Rett syndrome. A generous $85,000 donation from RSAM will support an ongoing clinical trial for Rettsyndrome.org at Boston Children's Hospital. The trial is funded by a Rettsyndrome.org ANGEL grant to Dr. Walter Kaufmann for a Phase 2b clinical study of IGF-1. (2015-08-12)

Rett Syndrome Association of MA joins Rettsyndrome.org to explore treatment option
Rett Syndrome Association of Massachusetts and Rettsyndrome.org are thrilled to announce today their renewed commitment to work together to defeat Rett syndrome. A generous $150,000 donation from RSAM will support a Rettsyndrome.org approved grant to Dr. Michela Fagiolini's project at the Boston Children's Hospital: 'Assessing NMDA receptor (NMDAr) modulators to ameliorate cortical regression in Rett syndrome'. (2014-08-06)

Discovered a cause of mental retardation and autism
The term intellectual disability covers a large number of clinical entities, some with known cause and others of uncertain origin. For example Down syndrome is due to an extra copy of chromosome 21 and Rett syndrome is in part caused by a mutation in the control switch gene called MeCP2. (2015-07-20)

Discovery will aid identification of misregulated genes in Rett Syndrome
Adrian Bird of the University of Edinburgh and colleagues report today in the online issue of Molecular Cell that the (2005-09-02)

International Rett Syndrome Foundation recognizes Dr. Huda Zoghbi, Dr. Alan Percy
The International Rett Syndrome Foundation presented its most prestigious awards to Dr. Huda Zoghbi of BCM and Dr. Alan Percy of University of Alabama-Birmingham. (2009-06-02)

Discovery fuels hope for Rett syndrome treatment
Vanderbilt University researchers have relieved symptoms of Rett syndrome in a mouse model with a small molecule that works like the dimmer switch in an electrical circuit. (2017-08-23)

OHSU discovery may lead to new treatment for Rett Syndrome
Researchers at Oregon Health & Science University have discovered that a molecule critical to the development and plasticity of nerve cells - brain-derived neurotrophic factor -- is severely lacking in brainstem neurons in mutations leading to Rett syndrome, a neurological developmental disorder. The finding has implications for the treatment of neurological disorders, including Rett syndrome that affects one in 10,000 baby girls. (2012-01-27)

Introduction of the 'Rett protein' in post-mitotic neurons rescues Rett Syndrome in mice
Rudolf Jaenisch of the Whitehead Institute of Biomedical Research in Cambridge, MA reports in the April 6, 2004 online issue of the Proceedings of the National Academy of Sciences that introduction of the MeCP2 protein into post-mitotic nerve cells of MeCP2 mutant mice rescues the symptoms of Rett Syndrome. This raises the possibility that neurons are functionally normal in a newborn child and that neural dysfunction manifests itself only later due to prolonged MeCP2 deficiency. (2004-04-07)

MIT: A possible treatment for Rett syndrome
A molecule that promotes brain development could serve as a possible treatment for Rett syndrome, the most common form of autism in girls, according to researchers at MIT's Picower Institute for Learning and Memory and the Whitehead Institute for Biomedical Research. (2009-02-09)

Researchers discover an alternate form for the disease gene associated with Rett syndrome
Researchers at Toronto's Hospital for Sick Children and their collaborators have identified a new form of the protein MECP2 which regulates the complex expression of Rett syndrome and other neurological disorders including autism, childhood schizophrenia and some forms of mental retardation. This discovery is being incorporated into a new molecular test that will aid not only the diagnosis of Rett syndrome, but also for other developmental disabilities. This research is reported in the April issue of the journal Nature Genetics. (2004-03-22)

Researchers alleviate symptoms of Rett syndrome in mice
Rett syndrome is a debilitating neurological disorder occurring primarily in girls. While some existing therapies might ease particular symptoms of the condition, there is no current way to address the syndrome at a molecular level. Now, researchers at Whitehead Institute for Biomedical Research, in collaboration with scientists at Brandeis University, have dramatically reduced certain manifestations of Rett Syndrome in mice, marking a clear path in which to explore possible therapies for people. (2006-02-01)

Researchers reverse symptoms in neurologic disease model
Rett syndrome is a devastating genetic, neurologic disorder that typically affects girls, resulting in severe disability and often accompanied by autistic behavior. Most Rett patients will live into middle age and require specialized full-time care. There is no cure, but researchers from Case Western Reserve University School of Medicine have been working to find ways to restore brain function and reverse disabilities associated with Rett syndrome. (2018-01-29)

Rettsyndrome.org announces new 2015 Translational Research Awards
Rettsyndrome.org announces today that the Board of Trustees has awarded $1M to launch the Read-Through Program, further translational research in the area of neuro-habilitation, and fund clinical research. (2015-06-25)

IRSF awards over $650,000 for Translational Rett Syndrome research
The International Rett Syndrome Foundation (IRSF) announced today that it is awarding over $650,000 to support eight cutting-edge projects that aim to accelerate translational research to develop treatments for Rett syndrome. IRSF is the world's largest private source of funding for biomedical and clinical Rett syndrome research. Since 1998, IRSF has cumulatively funded over $24M in high quality, peer-reviewed basic and translational research grants that have significantly advanced Rett syndrome research towards finding a cure. (2011-08-18)

Rettsyndrome.org invests $1.5 million in new 2014 awards
The International Rett Syndrome Foundation now doing business as Rettsyndrome.org announces today that the Board of Trustees has awarded $1.5 million to support 10 new grants to further translational research and launch of the neuro-habilitation therapeutic program, and fund clinical research. (2014-08-18)

IRSF announces funding of first clinical trial with disease-modifying therapy for Rett syndrome
The International Rett Syndrome Foundation announced that it will provide $200,000 to support a newly proposed clinical trial with a growth-factor based treatment for Rett syndrome. The study will be conducted by a team of clinical trials specialists at Boston Children's Hospital, led by Dr. Omar Khwaja, M.D., Ph.D., and will be the first potentially disease modifying therapy ever to be tested in Rett syndrome patients. (2009-09-21)

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