Scientists reverse a sensory impairment in mice with autism
Using a genetic technique that allows certain neurons in the brain to be switched on or off, UCLA scientists reversed a sensory impairment in mice with symptoms of autism, enabling them to learn a sensory task as quickly as healthy mice.Thefindings, which appear in the journal Nature Neuroscience, offer an intriguing glimpse of a potential strategy to help people with autism make sense of what their eyes see.In humans, the ability to perceive visual information is critical to learning of all kinds, including the interpretation of social cues. In children with autism, avoiding eye contact and struggling to understand people...
Source: UCLA Newsroom: Health Sciences - September 24, 2018 Category: Universities & Medical Training Source Type: news

Penn researchers: Class of neurological disorders share 3D genome folding pattern
(University of Pennsylvania) Researchers at the University of Pennsylvania have found a new common thread linking nearly all of the trinucleotide repeat expansion diseases, which include ALS, Huntington's Disease and Fragile X Syndrome, involving the complicated 3D patterns that the DNA is folded into in order to fit in the nucleus of the cell. Nearly all of the short tandem repeats known to grow unstable in disease are located at the boundaries that separate neighboring folded domains. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 18, 2018 Category: International Medicine & Public Health Source Type: news

Turning up translation in fragile X syndrome
(Source: ScienceNOW)
Source: ScienceNOW - August 16, 2018 Category: Science Authors: Aryal, S., Klann, E. Tags: Development perspective Source Type: news

Fragile X mental retardation 1 gene enhances the translation of large autism-related proteins
Mutations in the fragile X mental retardation 1 gene (FMR1) cause the most common inherited human autism spectrum disorder. FMR1 influences messenger RNA (mRNA) translation, but identifying functional targets has been difficult. We analyzed quiescent Drosophila oocytes, which, like neural synapses, depend heavily on translating stored mRNA. Ribosome profiling revealed that FMR1 enhances rather than represses the translation of mRNAs that overlap previously identified FMR1 targets, and acts preferentially on large proteins. Human homologs of at least 20 targets are associated with dominant intellectual disability, and 30 ot...
Source: ScienceNOW - August 16, 2018 Category: Science Authors: Greenblatt, E. J., Spradling, A. C. Tags: Development reports Source Type: news

Fragile X: New drug strategy corrects behavior/biochemical measures in mouse model
(Emory Health Sciences) Research in mice shows that a pharmacological strategy can alleviate multiple behavioral and cellular deficiencies in a mouse model of fragile X syndrome, the most common inherited form of intellectual disability. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 13, 2018 Category: International Medicine & Public Health Source Type: news

CRISPR-Cas9 Gene Editing Reduces Repetitive Behavior In Mice With Autism-Like Syndrome
researchers have utilized CRISPR-Cas9 gene editing in mice with a form of Fragile X Syndrome to alter gene expression, resulting in reduction of repetitive behaviors. Fragile X Syndrome (FXS) is the most commonly identified single-gene cause of autism spectrum disorder (ASD). (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - June 25, 2018 Category: Pharmaceuticals Authors: Robert Glatter, MD, Contributor Source Type: news

CRISPR editing reduces repetitive behavior in mice with a form of autism
(University of California - Berkeley) Mice with fragile X syndrome are a common model for autism because the mice exhibit exaggerated repetitive behavior typical of the disorder. UC Berkeley and UT San Antonio researchers have for the first time gotten CRISPR into the brain to successfully edit a gene and reduce repetitive behavior. The CRISPR-Gold technique knocked out a gene for a neurotransmitter receptor, damping overexcitation and associated burying behavior. The technique could allow CRISPR therapies for other brain disorders. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 25, 2018 Category: International Medicine & Public Health Source Type: news

Examining the influence of social-environmental variables on self-injurious behaviour in adolescent boys with fragile X syndrome - Hall SS, Hustyi KM, Barnett RP.
BACKGROUND: Individuals with fragile X syndrome (FXS), the most common known inherited form of intellectual disability, are at increased risk for showing specific forms of self-injurious behaviour (SIB) such as hand biting and head hitting, suggesting that... (Source: SafetyLit)
Source: SafetyLit - April 30, 2018 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news

White Matter Tracts Differ for Infants With Fragile X Syndrome
Significant differences identified in the development of 12 fiber tracts between FXS, controls (Source: The Doctors Lounge - Psychiatry)
Source: The Doctors Lounge - Psychiatry - April 9, 2018 Category: Psychiatry Tags: Neurology, Pathology, Pediatrics, Psychiatry, Journal, Source Type: news

White Matter Tracts Differ for Infants With Fragile X Syndrome
MONDAY, April 9, 2018 -- There are significant differences in the development of 12 fiber tracts between infants with fragile X syndrome (FXS) and controls, according to a study published online April 4 in JAMA Psychiatry. Meghan R. Swanson, Ph.D.,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 9, 2018 Category: Pharmaceuticals Source Type: news

Main Line pharma company plans key study of Fragile X syndrome therapy
Zynerba Pharmaceuticals said Monday it plans — with the Food and Drug Administration's approval — to initiate a single pivotal study later this year to support a new drug application for its experimental cannabis-based treatment for Fragile X Syndrome. Fragile X is a rare genetic developmental disability that is a leading known cause of b oth inherited intellectual disability and autism spectrum disorder. It affects an estimated one in 3,600 to 4,000 males and one in 4,000 to 6,000 females.… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 5, 2018 Category: Health Management Authors: John George Source Type: news

Fragile X syndrome neurons restored using CRISPR/Cas9-guided activation strategy
(Whitehead Institute for Biomedical Research) Fragile X syndrome is the most frequent cause of intellectual disability in males, affecting 1 out of 3600 boys born. For the first time, researchers at Whitehead Institute have restored activity to the fragile X syndrome gene in affected neurons using a modified CRISPR/Cas9 system that removes the methylation--the molecular tags that keep the mutant gene shut off--suggesting that this method may be useful for targeting diseases caused by abnormal methylation. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - February 21, 2018 Category: International Medicine & Public Health Source Type: news

Finding unravels nature of cognitive inflexibility in Fragile X syndrome
(New York University) Mice with the genetic defect that causes Fragile X syndrome (FXS) learn and remember normally, but show an inability to learn new information that contradicts what they initially learned, shows a new study by a team of neuroscientists. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 22, 2018 Category: International Medicine & Public Health Source Type: news

Functional changes of AMPA responses in human induced pluripotent stem cell-derived neural progenitors in fragile X syndrome
We examined functional maturation of the excitatory transmission system in FXS by investigating the response of FXS patient–derived neural progenitor cells to the glutamate analog (AMPA). Neural progenitors derived from induced pluripotent stem cell (iPSC) lines generated from boys with FXS had augmented intracellular Ca2+ responses to AMPA and kainate that were mediated by Ca2+-permeable AMPA receptors (CP-AMPARs) lacking the GluA2 subunit. Together with the enhanced differentiation of glutamate-responsive cells, the proportion of CP-AMPAR and N-methyl-d-aspartate (NMDA) receptor–coexpressing cells was increas...
Source: Signal Transduction Knowledge Environment - January 16, 2018 Category: Science Authors: Achuta, V. S., Möykkynen, T., Peteri, U.-K., Turconi, G., Rivera, C., Keinänen, K., Castren, M. L. Tags: STKE Research Articles Source Type: news

What Products Will Ultimately Win in Diagnostics?
It turns out there are actually several classes of diagnostic technologies that seem like they are really hot right now, but are actually overhyped, according to a panel of laboratory experts who addressed the subject during the recent J.P. Morgan Healthcare Conference in San Francisco, CA. Perhaps one of the hottest topics that have emerged in diagnostics over the past few years is liquid biopsy and while that technology is finding its way into hospital laboratories for certain applications, Nick Papadopoulos, a professor at Johns Hopkins University, said that in early detection of cancer, at least, liquid biopsy is ...
Source: MDDI - January 15, 2018 Category: Medical Devices Authors: Amanda Pedersen Tags: IVD Source Type: news

Main Line pharma company developing synthetic cannabinoid therapies narrows its focus
The companies will concentrate on developing treatments for Fragile X syndrome, epilepsy, Tourette's and other neurological and psychiatric disorders. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - January 4, 2018 Category: Health Management Authors: John George Source Type: news

Brainstem changes underlie sound sensitivity in fragile X mouse model
(Society for Neuroscience) Developmental changes in the brainstem driven by fragile X syndrome (FXS) -- a condition that often co-occurs with autism spectrum disorder in humans -- may underlie the hypersensitivity to sound observed in both humans and a mouse model of the disorder, according to animal research published in eNeuro. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 26, 2017 Category: International Medicine & Public Health Source Type: news

Study finds link between fragile X syndrome gene and dysregulated tissue growth
(Indiana University) A study led by Indiana University researchers found a previously undetected link between the gene that causes fragile X syndrome and increased tissue growth in the intestines of fruit flies modified to model the disease. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - December 5, 2017 Category: International Medicine & Public Health Source Type: news

Selective PDE4D inhibitor shows potential to treat Fragile X autism spectrum disorder
(Kureczka/Martin Associates) New research suggests the potential utility of BPN14770, a selective PDE4D inhibitor, in the treatment of Fragile X syndrome (FXS) and possibly other autism spectrum disorders. Daily BPN14770 dosing in a mouse Fragile X model showed reduce hyperarousal, improved social interactions and natural behaviors, as well as changes in neuronal dendrite structure. BPM14770 is currently in clinical testing by Tetra Discovery Partners as a prospective treatment for memory and cognitive problems associated with Alzheimer's disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 7, 2017 Category: International Medicine & Public Health Source Type: news

Reducing eIF4E-eIF4G interactions restores the balance between protein synthesis and actin dynamics in fragile X syndrome model mice
Fragile X syndrome (FXS) is the most common form of inherited intellectual disability and autism spectrum disorder. FXS is caused by silencing of the FMR1 gene, which encodes fragile X mental retardation protein (FMRP), an mRNA-binding protein that represses the translation of its target mRNAs. One mechanism by which FMRP represses translation is through its association with cytoplasmic FMRP-interacting protein 1 (CYFIP1), which subsequently sequesters and inhibits eukaryotic initiation factor 4E (eIF4E). CYFIP1 shuttles between the FMRP-eIF4E complex and the Rac1–Wave regulatory complex, thereby connecting translati...
Source: Signal Transduction Knowledge Environment - November 7, 2017 Category: Science Authors: Santini, E., Huynh, T. N., Longo, F., Koo, S. Y., Mojica, E., DAndrea, L., Bagni, C., Klann, E. Tags: STKE Research Articles Source Type: news

Aberrant Rac1-cofilin signaling mediates defects in dendritic spines, synaptic function, and sensory perception in fragile X syndrome
Fragile X syndrome (FXS) is the most common inherited cause of intellectual disabilities and a leading cause of autism. FXS is caused by a trinucleotide expansion in the gene FMR1 on the X chromosome. The neuroanatomical hallmark of FXS is an overabundance of immature dendritic spines, a factor thought to underlie synaptic dysfunction and impaired cognition. We showed that aberrantly increased activity of the Rho GTPase Rac1 inhibited the actin-depolymerizing factor cofilin, a major determinant of dendritic spine structure, and caused disease-associated spine abnormalities in the somatosensory cortex of FXS model mice. Inc...
Source: Signal Transduction Knowledge Environment - November 7, 2017 Category: Science Authors: Pyronneau, A., He, Q., Hwang, J.-Y., Porch, M., Contractor, A., Zukin, R. S. Tags: STKE Research Articles Source Type: news

Neuronal activity drives FMRP- and HSPG-dependent matrix metalloproteinase function required for rapid synaptogenesis
Matrix metalloproteinase (MMP) functions modulate synapse formation and activity-dependent plasticity. Aberrant MMP activity is implicated in fragile X syndrome (FXS), a disease caused by the loss of the RNA-binding protein FMRP and characterized by neurological dysfunction and intellectual disability. Gene expression studies in Drosophila suggest that Mmps cooperate with the heparan sulfate proteoglycan (HSPG) glypican co-receptor Dally-like protein (Dlp) to restrict trans-synaptic Wnt signaling and that synaptogenic defects in the fly model of FXS are alleviated by either inhibition of Mmp or genetic reduction of Dlp. We...
Source: Signal Transduction Knowledge Environment - November 7, 2017 Category: Science Authors: Dear, M. L., Shilts, J., Broadie, K. Tags: STKE Research Articles Source Type: news

Special issue: Fragile X syndrome
In this issue of Science Signaling, three papers uncover molecular mechanisms of neurological dysfunction in animal models of fragile X syndrome. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - November 7, 2017 Category: Science Authors: Ferrarelli, L. K. Tags: STKE Editors ' Choice Source Type: news

Positive Topline Results for Cannabinoid Gel in Fragile X Positive Topline Results for Cannabinoid Gel in Fragile X
An investigational transdermal cannabinoid gel led to significant and clinically meaningful improvement in anxiety and difficult behaviors in a phase 2 study of children with fragile X syndrome.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 29, 2017 Category: Consumer Health News Tags: Psychiatry News Source Type: news

Why Zynerba's stock soared 62% this week
Zynerba's stock price closed up 62 percent Thursday, at $9.44 per share, after the specialty pharmaceutical company posted positive study results from a midstage clinical trial of its experimental treatment for a rare developmental disorder. The Devon company is attempting to develop synthetic cannabinoid-based therapies for a variety of disorders. Zynerba said in a study of its ZYN002 cannabidiol gel in pediatric and adolescent patients with Fragile X syndrome that the new drug candidates met… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - September 29, 2017 Category: American Health Authors: John George Source Type: news

Zynerba's stock on a high as cannabis-based drug succeeds in trial
(Reuters) - Zynerba Pharmaceuticals Inc said on Thursday its cannabis-based gel met the main goal in a mid-stage study of patients with Fragile X syndrome, a genetic disorder that causes learning disabilities in adolescents and children. (Source: Reuters: Health)
Source: Reuters: Health - September 28, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Zynerba's cannabis-based gel meets main goal in mid-stage study
(Reuters) - Zynerba Pharmaceuticals Inc said on Thursday its cannabis-based gel achieved the main goal in a mid-stage study of patients with Fragile X syndrome, a genetic disorder that causes learning disabilities. (Source: Reuters: Health)
Source: Reuters: Health - September 28, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Zynerba's cannabis-based gel succeeds in mid-stage study
(Reuters) - Zynerba Pharmaceuticals Inc said on Thursday its cannabis-based gel succeeded in a mid-stage study of patients with Fragile X syndrome, a genetic disorder that causes learning disabilities. (Source: Reuters: Health)
Source: Reuters: Health - September 28, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

New role for fragile X protein could offer clues for treatment
(Howard Hughes Medical Institute) The protein behind fragile X syndrome, a leading cause of autism and intellectual disability, controls a suite of genetic regulators. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 28, 2017 Category: International Medicine & Public Health Source Type: news

New research on Fragile X syndrome reinforces importance of early detection
(Vanderbilt University) New insights into the long-lasting effects of Fragile X syndrome on connections in the brain during early development highlight the importance of early detection and treatment. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 25, 2017 Category: International Medicine & Public Health Source Type: news

Children with fragile X syndrome have a bias toward threatening emotion
(Elsevier) Anxiety occurs at high rates in children with fragile X syndrome (FXS), the most common form of inherited intellectual disability. Children with co-occurring anxiety tend to fare worse, but it can be hard to identify in infants. A new study in Biological Psychiatry: Cognitive Neuroscience and Neuroimaging reports that infants and children with FXS show bias toward threatening emotion, rather than positive emotion, a pattern highly linked with anxiety. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - August 23, 2017 Category: International Medicine & Public Health Source Type: news

Insight into learning via 'friend of fragile X' gene
(Emory Health Sciences) Fragile X syndrome, caused by a disruption of the gene FMR1, is the most common inherited form of intellectual disability. Emory scientists have revealed new aspects of the function of FMR1 and a 'friend' -- called ZC3H14 in humans/mice and dNab2 in flies. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 9, 2017 Category: International Medicine & Public Health Source Type: news

A therapy for FXS?
The antidiabetic drug metformin might be repurposed to treat patients with fragile X syndrome. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - July 4, 2017 Category: Science Authors: Ferrarelli, L. K. Tags: STKE Editors ' Choice Source Type: news

UCLA Medical Center doctor under investigation in organ harvesting case
(Natural News) Little Cole Hartman was dealt a bad hand right from the beginning. Born with fragile X syndrome, he had both physical and mental disabilities. When his mom died while he was just a toddler, he was adopted by Jeremy Hartman and his wife Elizabeth. Then, when he was just 8 years old, his... (Source: NaturalNews.com)
Source: NaturalNews.com - June 15, 2017 Category: Consumer Health News Source Type: news

Mice can ’t ‘tune out’ stimuli, mimicking sensory hypersensitivity in humans
This study ’s other authors are Daniel Cantu, Anubhuti Goel, Shilpa Mantri and William Zeiger, all of UCLA.The study was funded by a Paul and Daisy Soros Fellowship for New Americans and an NIH NINDS F30 Fellowship (NS093719); UCLA Neural Microcircuits training grant (T32-NS058280); a Eugene V. Cota-Robles Fellowship; the UCLA Medical Scientist Training Program (NIH NIGMS training grant GM08042; and a Developmental Disabilities Translational Research Program grant (20160969) from the John Merck Fund; a SFARI grant from the Simons Foundation (295438); and NIH NICHD grant (RO1 HD054453). (Source: UCLA Newsroom: Health Sciences)
Source: UCLA Newsroom: Health Sciences - June 12, 2017 Category: Universities & Medical Training Source Type: news

Animal models can't 'tune out' stimuli, mimicking sensory hypersensitivity in humans
(University of California - Los Angeles) Mice genetically engineered to mimic a type of autism in humans, fragile X syndrome, are unable to adapt to, or tune out, repeated stimulation to their whiskers -- unlike ordinary mice. The findings have implications for a common symptom -- sensory hypersensitivity -- in humans with autism. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 12, 2017 Category: International Medicine & Public Health Source Type: news

Better outcome measures needed for clinical trials for Fragile X Syndrome
(Johns Hopkins Medicine) A group of researchers from several institutions in the USA, including Johns Hopkins Medicine, reports that its review of 22 clinical trials of fragile X syndrome (FXS) suggests the need for a wider use of newer and improved treatment outcome measurement tools for this and other several neurodevelopmental disorders. FXS is the most common inherited form of intellectual disability and the most common form of autism associated with a single gene mutation. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - June 12, 2017 Category: International Medicine & Public Health Source Type: news

Diabetes drug may help symptoms of autism-associated condition
Metformin, the most widely used drug to treat type 2 diabetes, could potentially be used to treat symptoms of Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - May 15, 2017 Category: Science Source Type: news

Medical News Today: Metformin shows promise as a potential treatment for fragile X syndrome
New study shows that treatment with the common type 2 diabetes drug metformin improves social, behavioral, and neuron deficits in mouse model of fragile X. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - May 15, 2017 Category: Consumer Health News Tags: Autism Source Type: news

Diabetes drug may help symptoms of autism-associated condition
Metformin, the most widely used drug to treat type 2 diabetes, could potentially be used to treat symptoms of Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism. A new study led by researchers at McGill University, the University of Edinburgh and Université de Montréal has found that metformin improves social, behavioural and morphological defects in Fragile X mice. (Source: World Pharma News)
Source: World Pharma News - May 15, 2017 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Diabetes drug may help symptoms of autism associated condition
(University of Edinburgh) A widely used diabetes medication could help people with a common inherited form of autism, research shows. Scientists found that a drug called metformin improves sociability and reduces symptomatic behaviors in adult mice with a form of Fragile X syndrome. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - May 15, 2017 Category: International Medicine & Public Health Source Type: news

How Fragile X syndrome disrupts perception
A new study sheds light on the neural mechanisms of Fragile X syndrome. This genetic disorder, which affects males twice as often as females due to males'single X chromosome, causes disruptions in the way neurons transmit information to each other. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - May 5, 2017 Category: Science Source Type: news

Scientists gain insights into how Fragile X syndrome disrupts perception
(VIB (the Flanders Institute for Biotechnology)) A collaboration between scientists in Belgium, the United States, Norway, France and the UK has resulted in a study that sheds light on the neural mechanisms of Fragile X syndrome. This genetic disorder, which affects males twice as often as females due to males' single X chromosome, causes disruptions in the way neurons transmit information to each other. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 5, 2017 Category: Global & Universal Source Type: news

Finding drugs for fragile X syndrome
(Source: ScienceNOW)
Source: ScienceNOW - May 4, 2017 Category: Science Authors: Ferrarelli, L. K. Tags: twis Source Type: news

New connections: Treating fragile X syndrome
An in vivo drug screen can identify inhibitors of the signaling pathway that is activated in fragile X syndrome. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - May 2, 2017 Category: Science Authors: Ferrarelli, L. K. Tags: STKE Editors ' Choice Source Type: news

Hyperactive locomotion in a Drosophila model is a functional readout for the synaptic abnormalities underlying fragile X syndrome
We report that larval locomotion is augmented in a Drosophila FXS model. Genetic or pharmacological intervention on the BMPR2-LIMK pathway ameliorated the synaptic abnormality and locomotion phenotypes of FXS larvae, as well as hyperactivity in an FXS mouse model. Our study demonstrates that (i) the BMPR2-LIMK pathway is a promising therapeutic target for FXS and (ii) the locomotion phenotype of FXS larvae is a quantitative functional readout for the neuromorphological phenotype associated with FXS and is amenable to the screening novel FXS therapeutics. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - May 2, 2017 Category: Science Authors: Kashima, R., Redmond, P. L., Ghatpande, P., Roy, S., Kornberg, T. B., Hanke, T., Knapp, S., Lagna, G., Hata, A. Tags: STKE Research Resources Source Type: news

Ovid commences Phase I trial of OV101 to treat Angelman / Fragile X syndromes
Biopharmaceutical firm Ovid Therapeutics has commenced the Phase I clinical trial of OV101 for the treatment of adolescents with Angelman syndrome or Fragile X syndrome. (Source: Drug Development Technology)
Source: Drug Development Technology - April 10, 2017 Category: Pharmaceuticals Source Type: news

[Editors' Choice] Impaired phagocytosis in Fragile X
Defects in phagocytosis underlie both neurological and immunological symptoms in a fly model of Fragile X syndrome. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - April 4, 2017 Category: Science Authors: Nancy Gough (mailto:ngough at aaas.org) Source Type: news

Vencerx Therapeutics Announces Appointments of Robert H. Ring, Ph.D. as Chief Executive Officer and Jeffrey Stevenson as Chairman of the Board
NEW YORK, March 31, 2017 -- (Healthcare Sales & Marketing Network) -- Vencerx Therapeutics, a company focused on advancing treatments for neurodevelopmental disorders, today announced the appointments of Robert H. Ring, Ph.D. as Chief Executive Officer an... Biopharmaceuticals, Personnel Vencerx Therapeutics, Fragile X Syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 31, 2017 Category: Pharmaceuticals Source Type: news

Unique Characteristics of the X Chromosome and Related Disorders
This article reviews these unique characteristics and applies their guiding principles in a broad overview to Klinefelter syndrome, ornithine transcarbamylase (OTC) deficiency, hemophilia, and fragile X syndrome. (Source: NeoReviews recent issues)
Source: NeoReviews recent issues - March 31, 2017 Category: Pediatrics Authors: Shah, K., DeRemigis, A., Hageman, J. R., Sriram, S., Waggoner, D. Tags: Pediatric Drug Labeling Update Articles Source Type: news