Tag Archives: Core Aspects

Question?: Pdd Nos Symptoms

George asks…

Poll: Do you think vaccines cause or contribute to autism?

I’m curious to see what people really think.

admin answers:

No i dont believe they do whatsoever! I think parents are just being over cautious and flipping out for no reason. Vaccines have been around for ages, and now they are trying to say that it causes autisim? I dont think so. Dont you think there would have been more causes of that if it was actually linked to the vaccines. Its the same vaccine schedule, its not like the vaccines have changed much in the last 50 years.

Autism and autism spectrum disorders are complex neurodevelopmental disorders.Heritability contributes about 90% of the risk of a child developing autism, but the genetics of autism are complex and typically it is unclear which genes are responsible.Many other causes have been proposed, such as exposure of children to vaccines; these proposals are controversial and the vaccine hypotheses have no convincing scientific evidence.

Autism is a condition involving abnormalities of brain development and behavior which manifests itself before a child is three years old and has a steady course with no remission. It is characterized by impairments in social interaction and communication, as well as restricted and repetitive behavior. It is part of a larger family called the autism spectrum disorders (ASD) or pervasive developmental disorders (PDD), which include closely related syndromes such as Asperger syndrome and PDD-NOS. This article uses autism to denote the classic autistic disorder and ASD to denote the wider family.

Autism’s theory of causation is still incomplete. It has long been presumed that there is a common cause at the genetic, cognitive, and neural levels for autism’s characteristic triad of symptoms. However,here is increasing suspicion among researchers that autism does not have a single cause, but is instead a complex disorder with a set of core aspects that have distinct causes. Although these distinct causes have been hypothesized to often co-occur, it has also been suggested that the correlation between the causes has been exaggerated.The number of people known to have autism has increased dramatically since the 1980s, at least partly due to changes in diagnostic practice; it is unknown whether prevalence has increased as well. An increase in prevalence would suggest directing more attention and funding toward changing environmental factors instead of continuing to focus on genetics.

The consensus among mainstream autism researchers is that genetic factors predominate, but some are concerned, as one anonymous researcher put it, that “geneticists are running the show, and ignoring the environmental aspects.”Environmental factors that have been claimed to contribute to autism or exacerbate its symptoms, or may be important to consider in future research, include certain foods, infectious disease, heavy metals, solvents, diesel exhaust, PCBs, phthalates and phenols used in plastic products, pesticides, brominated flame retardants, alcohol, smoking, illicit drugs, and vaccines. Among these factors, vaccines have attracted much attention, as parents may first become aware of autistic symptoms in their child around the time of a routine vaccination, and parental concern about vaccines has led to a decreasing uptake of childhood immunizations and an increasing likelihood of measles outbreaks. However, as described in Mercury and MMR vaccine below, there is overwhelming scientific evidence showing no causal association between the measles-mumps-rubella vaccine and autism, and there is no scientific evidence that the vaccine preservative thiomersal helps cause autism.

In 2007 the National Institutes of Health announced an Autism Centers of Excellence (ACE) research program to find the causes of autism and identify new treatments for the disorder. Initial recipients are focusing on genetic factors, brain imaging, brain chemicals and functions including mirror neurons, effect on early parent-child behavior on autism, and learning in autistic children.
Http://en.wikipedia.org/wiki/Causes_of_autism

I am a mother of a 12 month old boy… Who is up to date on all his vaccines so far. And he will continue to get all his vaccinations. As so will any other children i should have. Im sorry but i wouldnt take the risk of my child actually contracting one of those disease just because some parents believe that it may be linked to autisim. Sorry, i would rather have my child vaccinated.

Powered by Yahoo! Answers

Research Published Supporting Disease-Modifying Potential Of STX209 For Fragile X Syndrome

Main Category: Autism
Also Included In: Genetics
Article Date: 21 Sep 2012 – 0:00 PDT Current ratings for:
Research Published Supporting Disease-Modifying Potential Of STX209 For Fragile X Syndrome
not yet ratednot yet rated
Seaside Therapeutics has announced the publication of two papers in Science Translational Medicine, supporting its lead candidate, STX209 (arbaclofen), for the treatment of fragile X syndrome (FXS). The works presented highlight STX209 as a potential disease-modifying drug in preclinical studies, with improvement in social function in a clinical trial of patients with FXS.

“In preclinical results, we demonstrate that STX209 treatment corrects core aspects of FXS pathophysiology in the fragile X mouse model, leading us to conclude that STX209 is a disease-modifying drug with the potential to significantly improve the lives of patients with fragile X,” said Randall Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. “In fact, in our Phase 2 clinical trial, we observed statistically significant improvements in social avoidance, a core symptom of fragile X syndrome that is characterized by behaviors such as preference to be alone and being withdrawn or isolated. Social avoidance is also a core symptom of autism spectrum disorders, suggesting STX209 could have a positive effect in this larger patient population. Together, these results support our continued development of STX209, which is currently enrolling patients in a Phase 3 study in fragile X syndrome and recently completed enrollment in a Phase 2b study in autism spectrum disorders.”

“There are currently no FDA approved therapeutics that address the core symptoms of fragile X syndrome, leaving patients and their caregivers with limited treatment options,” said Elizabeth Berry-Kravis, MD, PhD, Professor of Pediatrics, Neurology, and Biochemistry at Rush University Medical Center in Chicago, and the lead author of the paper on the Phase 2 study results. “We are very excited about the clinically meaningful improvements in social impairment observed to date in patients receiving STX209 – marking the first time a drug candidate has positively impacted a specific core symptom of fragile X. With continued success in ongoing clinical trials, I am hopeful that STX209 may be able to transform the treatment paradigm for fragile X syndrome – which would confirm what I have believed all along – that intellectual disability is not, as previously understood, an immutable condition.”

FXS is the most common inherited cause of intellectual disability (mental retardation) and the most prevalent known cause of autism. FXS is caused by the mutation of a single gene known as FMR1. FMR1 codes for a protein called fragile X mental retardation protein (FMRP) that is necessary for normal brain development. Without FMRP, patients exhibit several changes in their brains that underlie the cognitive and behavioral deficits associated with FXS. These changes include: alterations of neuronal morphology and an imbalance between excitatory and inhibitory neurotransmission in the brain. In addition, changes in localized protein synthesis have been observed in mouse models of FXS.

In the paper entitled “Postnatal pharmacological activation of the GABA-B receptor has potential to be disease-modifying in fragile X syndrome,” the researchers tested whether STX209 was effective in reversing the hallmark changes in the brains of mice lacking the Fmr1 gene. In vitro application of STX209 to the hippocampus of mice lacking Fmr1 reduced levels of localized protein synthesis to that of normal mice. Additionally, this treatment reduced a known functional consequence of increased protein synthesis, AMPA receptor internalization, to levels found in normal mice. Furthermore, in vivo administration of STX209 corrected alterations in neuronal morphology specific to Fmr1-deficient mice and FXS patients, but STX209 did not alter neuronal morphology in normal mice and STX209 also reduced protein translation in mice lacking Fmr1, but not in normal mice. Of note, the magnitude of effect observed with STX209 is comparable to that reported for selective mGluR5 antagonists. These results demonstrate that STX209 has the potential to modify the underlying disease of patients with FXS and support the development of STX209 in the clinic.*

The accompanying paper, “Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, Phase 2 trial,” detailed the results previously announced from the Company’s Phase 2 trial. The randomized, double-blind, placebo-controlled Phase 2 study was designed to evaluate the clinical effects of STX209 in 63 subjects, age 6-40 years, with a full mutation of the FMR1 gene. As previously reported, STX209 was well-tolerated and no metabolic side effects were observed. The most common adverse events in subjects receiving STX209 were upper respiratory infections (13%), sedation (8%) and headache (8%), compared to 10%, 2% and 2%, respectively, while receiving placebo. In per-protocol analyses, improvement was seen on the visual analog scale (VAS) ratings of parent-nominated problem behaviors and positive trends were observed on multiple global measures. While improvement was noted on the Irritability subscale of the Aberrant Behavior Checklist (ABC-I), the study’s primary endpoint, the magnitude was comparable to that observed on placebo and did not achieve statistical significance.

Social avoidance is a core symptom of FXS. Post-hoc analysis of ABC-Social Avoidance scale, a newly validated scale for the assessment of FXS, revealed a significant beneficial treatment effect in the full study population (p<0.01). A post-hoc subgroup of 27 subjects with more severe social impairment showed improvements on the Vineland II-Socialization raw score (p=0.03), the ABC-Social Avoidance scale (p=0.04) and on all global measures. The results suggest that STX209 has the potential to improve social function and behavior in patients with FXS.**

About STX209:

STX209 is an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist. Pathologies observed in certain neurodevelopmental disorders, including autism spectrum disorders and fragile X syndrome are believed to be caused by excessive activation of glutamate receptors and abnormally high ratios of excitatory to inhibitory neurotransmission in the brain. GABA-B receptors play an important role in modulating the release of glutamate and optimizing the ratio of excitatory to inhibitory neurotransmission. STX209 has demonstrated efficacy in preclinical models, suggesting that it may improve function in individuals with autism spectrum disorders and fragile X syndrome. With STX209, Seaside has successfully completed the largest, randomized, blinded, placebo-controlled trial (Phase 2) in patients with fragile X syndrome and an open-label Phase 2a exploratory trial in patients with autism spectrum disorders. Two Phase 3 studies, one in adolescents and adults (ages 12 to 50) and one in children (ages 5 to 11) with fragile X syndrome, are currently enrolling participants. A Phase 2b study in children, adolescents and adults (ages 5 to 21) with autism spectrum disorders recently completed enrollment.

Roche and Seaside Therapeutics recently entered into an alliance under which Roche may exercise options to commercialize Seaside’s arbaclofen (STX209) upon completion of certain clinical development phases in Fragile X Syndrome and Autism Spectrum Disorders, while Seaside will continue to lead the clinical development of these programs.

About Fragile X Syndrome:

Fragile X syndrome is a neurodevelopmental disorder characterized by impaired social function, cognition and speech, as well as attention deficits and low functional independence. It is the most common inherited form of intellectual disability and affects roughly 100,000 individuals in the U.S. It is also the largest known cause of autism. Fragile X syndrome is caused by a mutation of a single gene, the Fragile X mental retardation 1 (FMR1) gene, on the X chromosome. The FMR1 gene produces a protein needed for normal brain development. Individuals with fragile X syndrome lack this protein and, as a result, the majority of affected individuals will have significant intellectual disabilities and require life-time care. To date, there are no approved treatments for this disorder. The FDA and EMA have designated fragile X syndrome as an orphan disease.

Article adapted by Medical News Today from original press release. Click ‘references’ tab above for source.
Visit our autism section for the latest news on this subject. Study authors included Christina Henderson, Aileen M. Healy, Matthew Shumway, Rebecca S. Hammond, Friso R. Postima, Christopher Brynczka, Roger Rush and Randall L. Carpenter of Seaside Therapeutics; Lasani Wijetunge and Peter C. Kind of the University of Edinburgh; Mika Nakamoto Kinoshita and Stephen T. Warren of Emory University; Alicia Thomas and Richard Paylor of Baylor College of Medicine; Peter W. Vanderklish of The Scripps Research Institute; and Mark F. Bear of the Massachusetts Institute of Technology.
Study authors included Elizabeth M. Berry-Kravis of Rush University Medical Center, Randi J. Hagerman, David R. Hessl, Yi Mu and Danh V. Nguyen of the University of California Davis; Barbara Rathmell, Peter Zarevics, Maryann Cherubini, Karen Walton-Bowen, Paul P. Wang and Randall L. Carpenter of Seaside Therapeutics; Joseph Gonzalez-Heydrich of Children’s Hospital Boston; and Mark F. Bear of the Massachusetts Institute of Technology.
MacDougall Biomedical Communications, Inc.
Seaside Therapeutics Please use one of the following formats to cite this article in your essay, paper or report:

MLA

n.p. “Research Published Supporting Disease-Modifying Potential Of STX209 For Fragile X Syndrome.” Medical News Today. MediLexicon, Intl., 21 Sep. 2012. Web.
26 Sep. 2012. APA

Please note: If no author information is provided, the source is cited instead.


‘Research Published Supporting Disease-Modifying Potential Of STX209 For Fragile X Syndrome’

Please note that we publish your name, but we do not publish your email address. It is only used to let you know when your message is published. We do not use it for any other purpose. Please see our privacy policy for more information.

If you write about specific medications or operations, please do not name health care professionals by name.

All opinions are moderated before being included (to stop spam)

Contact Our News Editors

For any corrections of factual information, or to contact the editors please use our feedback form.

Please send any medical news or health news press releases to:

Note: Any medical information published on this website is not intended as a substitute for informed medical advice and you should not take any action before consulting with a health care professional. For more information, please read our terms and conditions.


View the original article here