Main Line biopharm firm gets a boost from the FDA
The Food and Drug Administration has granted Orphan Drug Designation to an experimental therapy Marinus Pharmaceuticals is developing to treat Fragile X Syndrome.
That prompts two questions: What is Fragile X Syndrome, and how is the Radnor, Pennsylvania, biopharmaceutical company’s new drug candidate attempting to treat the disorder.
Fragile X syndrome is genetic condition that causes a range of developmental problems and symptoms, including cognitive i mpairment, learning disabilities and behavioral… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 3, 2017 Category: Pharmaceuticals Authors: John George Source Type: news
Marinus Pharmaceuticals Receives FDA Orphan Drug Designation for Ganaxolone to Treat Fragile X Syndrome
RADNOR, Pa., Jan. 03, 2017 -- (Healthcare Sales & Marketing Network) -- Marinus Pharmaceuticals, Inc. (MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announ... Biopharmaceuticals, Neurology, FDA Marinus Pharmaceuticals, ganaxolone, Fragile X Syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - January 3, 2017 Category: Pharmaceuticals Source Type: news
Neural stem cells serve as RNA highways too
Scientists have caught the first glimpse of molecules shuttling along a sort of highway running the length of neural stem cells, which are crucial to the development of new neurons. This new view has given them an intriguing clue that a protein deficient in Fragile X syndrome, an autism-related disorder that causes intellectual disability, is responsible for moving at least some of this molecular cargo up and down the stem cells. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - December 1, 2016 Category: Science Source Type: news
Making Sense of Sensory Processing Disorder
Our bodies are wired for sensory input. Touch, (which includes temperature, texture and pressure), taste, sound, sight and smell are the ways in which we explore the world, from the moment we enter it. When all systems are functioning, we experience a feedback loop. For example, if you were shivering from the cold and wanted to feel warmth, you might put on a cozy sweater or wrap a fleece blanket around your shoulders. Your body would likely respond by relaxing, followed by an emotional relief and perhaps even a sigh. The next time you felt chilly, you would remember what it took to remedy that sensation and follow throug...
Source: Psych Central - November 22, 2016 Category: Psychiatry Authors: Edie Weinstein, MSW, LSW Tags: Attention Deficit Disorder Autism / Asperger's Caregivers Children and Teens Genetics Parenting Asperger Syndrome Pervasive Developmental Disorder Sensory integration dysfunction Sensory Processing Disorder Source Type: news
Study implicates glial cells in fragile X syndrome
(University of California - Santa Cruz) Research on fragile X syndrome, the most common inherited cause of mental retardation, has focused mostly on how the genetic defect alters the functioning of neurons in the brain. A new study focusing on a different type of brain cell, the glial cells known as astrocytes, indicates they also are impaired by the genetic defect and are involved in the symptoms of the disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 4, 2016 Category: Global & Universal Source Type: news
New explanation offered for symptoms of fragile X syndrome
Until recently, scientists thought they understood one of the underlying causes of fragile X syndrome, the most common inherited cause of intellectual disability in the United States. The syndrome, which is associated with autism, was believed to be linked primarily to overactivity in a molecular pathway in the brain. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - September 21, 2016 Category: Science Source Type: news
New explanation offered for symptoms of fragile X syndrome
A new explanation for some of the symptoms of fragile X syndrome, the most common inherited cause of intellectual disability, has been proposed by researchers. Their explanation suggests new targets for treatment. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - September 20, 2016 Category: Science Source Type: news
Sertraline, brand named Zoloft, improves functioning in young children with fragile X
Treatment with sertraline may provide nominal but important improvements in cognition and social participation in very young children with fragile X syndrome, the most common genetic cause of intellectual disability and the leading single-gene cause of autism, a study has found. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - August 26, 2016 Category: Science Source Type: news
Simple, sensitive, and cost-effective assays for analyzing Fragile X-related disorders
Fragile X syndrome, the most common heritable cause of intellectual disability and a frequent cause of autism, is characterized by abnormalities of the FMR1 gene that are difficult to analyze. Preclinical studies of Fragile X and the Fragile X-related disorders are hampered by the lack of low-cost and sensitive yet simple methods. Researchers have now developed a set of assays that are robust, cheap enough for routine research use, and may be suitable for initial patient screening. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - August 12, 2016 Category: Science Source Type: news
Researchers at NIH have developed simple, sensitive, and cost-effective assays for analyzing Fragile X-related disorders
(Elsevier Health Sciences) Fragile X syndrome, the most common heritable cause of intellectual disability and a frequent cause of autism, is characterized by abnormalities of the FMR1 gene that are difficult to analyze. Preclinical studies of Fragile X and the Fragile X-related disorders are hampered by the lack of low-cost and sensitive yet simple methods. NIH researchers have now developed a set of assays that are robust, cheap enough for routine research use, and may be suitable for initial patient screening. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 12, 2016 Category: Global & Universal Source Type: news
Neuroscience study identifies new trigger mechanism for fragile X syndrome in mice
A new study identifies an astroglial trigger mechanism as contributing to symptoms of fragile X syndrome in mice. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - July 5, 2016 Category: Science Source Type: news
Neuroscience study identifies new trigger mechanism for fragile X syndrome in mice
(Tufts University, Health Sciences Campus) A study published today in the Journal of Neuroscience led by Yongjie Yang of Tufts University School of Medicine identifies an astroglial trigger mechanism as contributing to symptoms of fragile X syndrome in mice. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 5, 2016 Category: Global & Universal Source Type: news
[Research Article] Augmented noncanonical BMP type II receptor signaling mediates the synaptic abnormality of fragile X syndrome
Blocking a BMP signaling pathway may ameliorate neurological defects in patients with fragile X syndrome (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - June 7, 2016 Category: Science Authors: Risa Kashima, Sougata Roy, Manuel Ascano, Veronica Martinez-Cerdeno, Jeanelle Ariza-Torres, Sunghwan Kim, Justin Louie, Yao Lu, Patricio Leyton, Kenneth D. Bloch, Thomas B. Kornberg, Paul J. Hagerman, Randi Hagerman,
Giorgio Lagna, Akiko Hata Source Type: news
[Focus] BMP signaling turns up in fragile X syndrome: FMRP represses BMPR2
Reducing bone morphogenetic protein (BMP) signaling may reverse neuroanatomical defects in fragile X syndrome. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - June 5, 2016 Category: Science Authors: Heather T. Broihier Source Type: news
Study on fragile X syndrome uses fruitfly's point of view to identify new treatments
The hormone insulin - usually associated with diabetes -- is involved in the daily activity patterns and cognitive deficits in the fruitfly model of FXS. Results reveal a metabolic pathway that can be targeted by new and already approved drugs to treat fragile X patients. This syndrome is the most common genetically inherited cause of intellectual disability in humans. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - April 26, 2016 Category: Science Source Type: news
