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What is VSL#3? – Video

Posted: Published on December 19th, 2014

What is VSL#3? VSL#3 is a high potency probiotic medical food that is clinically proven in the dietary management of irritable bowel syndrome (IBS), ulcerative colitis (UC) and ileal pouch in adults and children.... By: VSL3Probiotic … Continue reading

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Parent Project Muscular Dystrophy and Solid Ventures Partner on Solid Suit, an Assistive Device for Duchenne Muscular …

Posted: Published on December 19th, 2014

From left to right: Stanley Nelson, MD, Center for DMD at UCLA; Thomas Sugar, PhD, Arizona State University Polytechnic School; Tom Egan, SRI International; Roy Kornbluh, SRI International; Laura Case, DPT, Duke University School of Medicine; Keith Van Houten; Annie Kennedy, PPMD; Matt Arnold, Solid Ventures; Lee Sweeney, PhD, University of Pennsylvania; Tina Duong, DPT, Children's National Medical Center; Annie Ganot; Ilan Ganot, Solid Ventures; Eytani Ganot; Andrey Zarur, PhD, Solid Ventures; Brenda Wong, MD, Comprehensive Neuromuscular ... Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) HACKENSACK, N.J., Dec. 18, 2014 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), a nonprofit organization dedicated to the fight to end Duchenne muscular dystrophy (Duchenne), announced today a collaboration with Solid Ventures (Solid) to engage SRI Internationalin the research and development of the "Solid Suit," a soft, wearable assistive device for people with Duchenne. Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 to 5,000 live male births. The loss of a key muscle protein called dystrophin causes muscle wasting and weakness, eventually leading to the loss of ambulation, difficulty breathing, and heart failure. Death typically occurs in the mid-to late 20s. The Solid … Continue reading

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Pfizer begins Phase II trial of PF-06252616 to treat duchenne muscular dystrophy

Posted: Published on December 19th, 2014

PBR Staff Writer Published 18 December 2014 Pfizer has started patient enrollment in a multicenter Phase II clinical trial of PF-06252616, an experimental, infused, anti-myostatin monoclonal antibody, to treat Duchenne muscular dystrophy (DMD). The trial is designed to evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged six to less than ten years old diagnosed with DMD regardless of genotype. DMD is genetic disorder characterized by progressive muscle degeneration and weakness. Myostatin is a naturally occurring protein that helps control muscle growth and it is believed that blocking its activity may have potential therapeutic application in treating muscle wasting diseases such as DMD. Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Kevin Lee said: "DMD is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwidei with no current treatment options. "We are pleased to be taking this important next step in the development of PF-06252616 as an investigational therapy for DMD in the hopes of potentially bringing a much-needed therapy to individuals and families with this devastating disease." Based on the proposed mechanism of action of PF-06252616, the company is exploring whether there is the potential to increase muscle mass and … Continue reading

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Why a miracle drug exists but you cant have it yet

Posted: Published on December 19th, 2014

Andy Extance, mosaicscience.com Posted: Thursday, December 18, 2014, 3:10 AM Had it begun five years earlier, Leo Le Gals story would have followed a depressingly familiar pattern. Aged somewhere between 8 and 11, Leo would lose the ability to walk and go into a wheelchair. In his teens, hed progressively lose the use of his arms and with them, his independence. And eventually, some time between the ages of 21 and 30, his heart and lungs would give out, causing him to suffocate. But today there is a tantalising hope. Leos parents, Ruth and Damien, got him onto a clinical trial for a drug that promises a reprieve. Its one of three drugs that are trying a totally new approach: directly treating the genetic problem that causes his disease, Duchenne muscular dystrophy (DMD), rather than simply treating its symptoms. Trials like Leos have sent parents scrambling to get their sons on them and have brought the drugs to the threshold of widespread availability. Yet if he werent already involved, the drug would probably remain out of reach for him, as it is for most of the 250,000 boys with DMD worldwide. Leos condition occurs because hes a boy. As a … Continue reading

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Clinical trials of cannabis-based epilepsy medicine to start in the UK

Posted: Published on December 19th, 2014

December 18, 2014 Lorraine Roberston with son Max, aged 12, who will be among the first UK patients to receive a medicine derived from the cannabis plant to treat his epilepsy condition. Dr. Richard Chin from the University of Edinburgh's Muir Maxwell Epilepsy Centre (right) is helping to lead the study. (Credit: Jane Barlow Photography) Chuck Bednar for redOrbit.com Your Universe Online UK health officials have given the go-ahead for doctors to test a new treatment derived from the cannabis plant in children suffering from certain forms of severe epilepsy. The new treatment is known as Epidiolex, and it does not contain the ingredient responsible for creating the high associated with recreational marijuana use, researchers from the University of Edinburgh explained. Instead, it is based off of one of the plants non-psychoactive components, cannabidiol (CBD), the health benefits of which have been examined in clinical trials. The university explained that early studies conducted in the US have indicated that some epileptic children treated with CBD could experience less frequent and less severe seizures. This will mark the first time that the treatment method has been tested in the UK, and it will involve a randomized controlled trial at the University … Continue reading

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Cannabis treatment for children

Posted: Published on December 19th, 2014

Children with severe epilepsy could be helped by a new treatment derived from the cannabis plant which is to be trialled in the UK. Doctors in the UK have been given the go-ahead to test the medicine, which does not contain the ingredient that produces the high associated with recreational cannabis use. The treatment - called Epidiolex - is based on one of the non-psychoactive components of the cannabis plant, called CBD. Early studies in the US have shown that treatment with CBD may reduce the frequency and severity of seizures in children with severe forms of epilepsy. The new trial marks the first time the treatment has been tested in the UK. Patients are being enrolled for a randomised controlled trial of the treatment at The University of Edinburgh's Muir Maxwell Epilepsy Centre, based at the Royal Hospital for Sick Children in Edinburgh, and Great Ormond Street Hospital. Dr Richard Chin, Director of the Muir Maxwell Epilepsy Centre, said: "Many children with serious forms of epilepsy do not respond to the medications that we currently have available. We need new means of treating these conditions so that we can give back some quality of life to these children and … Continue reading

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Autism and Intellectual Disability Studied in Quest for New Treatment Approaches

Posted: Published on December 19th, 2014

Contact Information Available for logged-in reporters only Newswise Some of the genetic diseases that can cause autism spectrum disorder (ASD) and intellectual disability (ID) are so rare that even physicians who specialize in treating them cant be certain they have seen every possible symptom. Thats a problem for those clinicians, and it also makes it difficult to design interventions in hopes of improving the lives of people who live with the diseases. But a new nationwide effort, the Developmental Synaptopathies Consortium (DSC), which includes Rush and other top medical centers, is seeking to change that. The DSC will link families and specialists scattered across the country in a study that could provide solid pictures of three rare diseases that, among other devastating effects, can cause ASD and ID. The DSC will carry out the five-year study at 10 medical centers, enlisting a total of 330 children ages 3 to 21 to develop a natural history of the progression of the diseases tuberous sclerosis complex, Phelan-McDermid Syndrome and PTEN Hamartoma Tumor Syndrome. These three rare genetic diseases all disrupt the formation of synapses in the brain. Rush Pediatric Neurologist Dr. Elizabeth Berry-Kravis and Clinical Psychologist Latha Soorya,PhD, will be co-investigators in … Continue reading

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New autism centre to open in 2016

Posted: Published on December 19th, 2014

MP Kellie Leitch (Minister of Status for Women), Deputy premier Rich Coleman, Olympian Silken Laumann, and GoodLife Fitness Founder David Patchell-Evans, were among those who took part in Tuesday afternoons groundbreaking on Sea Island of the new Pacific Autism Family Centre at the foot of Hudson Avenue. image credit: Martin van den Hemel As any parent of a child who has been diagnosed with autism knows, there are so many questions that just dont have answers. At a groundbreaking ceremony on Sea Island Tuesday afternoon, work began on a $33 million Pacific Autism Family Centre project that hopes to provide parents with answers and support, their children with treatment and care, while others work on cutting-edge research into the developmental disorder that impacts typical brain growth. Olympian Silken Laumann joined her husband, David Patchell-Evans, founder of GoodLife Fitness, at the event where they shared how autism has impacted their lives, through Patchell-Evans daughter Kilee. Whether it has been obstacles in business or the challenge of living with rheumatoid arthritis, Ive always felt I had some control over the outcomes, Patchell-Evans said. What I have not chosen, and at many times have felt overwhelmed by, is the devastation of being a … Continue reading

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Solving the Autism Puzzle

Posted: Published on December 19th, 2014

His name was David. He was 10 years old and, to put it bluntly, compellingly weirdespecially in the buttoned-down, groomed normality of suburban Long Island in the early 1960s. At the time, Michael Wigler was a ninth-grade student in Garden City, and he liked to hang out at the home of his girlfriend. Thats where he encountered David, her younger brother. Half a century later, he still cant get the boy out of his mind. He was just like from another planetit was like meeting an alien, says Wigler, who ended up a little further east on Long Island as a geneticist at Cold Spring Harbor Laboratory. He was so different from anybody I had ever met before. First of all, he threw his arms about a lot. And then he moved his head around a lot and would never look at you when he talked to you. And he had an uncanny knowledge of baseball statistics. And I just thought, you know, Boy, this guy is really different. I mean, hes not just a little different. Hes very different. In the 1950s and 1960s, children like David were pretty much anomalies without a name. Long after becoming a prominent … Continue reading

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Rochester General Hospital receives award

Posted: Published on December 19th, 2014

Updated: Thursday, December 18 2014, 06:35 PM EST Rochester General Hospital has received an award for its stroke treatment. RGH received the Get With The Guidelines Stroke Gold-Plus Quality Achievement Award from the American Heart Association/American Stroke Association. This recognition is earned by hospital teams who achieve specific quality measures identified as vital for the optimal treatment of stroke. Rochester General was also re-certified by the AHA/ASA as a Target: Stroke Honor Roll hospital, for continuing to meet quality goals aimed at reducing the time between a patient's arrival at the hospital and their receiving the clot-busting drug tPA, the only FDA-approved drug in the treatment of ischemic stroke. Patients who receive tPA within three hours of the onset of stroke symptoms are more likely to recover quickly and avoid severe disability. Two million brain cells are lost every minute during a stroke, making the speed at which effective treatment is administered a leading factor in the patient's chances of achieving a full recovery. For years, the Rochester General emergency department has treated more stroke patients than almost any upstate New York hospital. "Rochester General Hospital has long been known as a leader in developing processes that improve the quality … Continue reading

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