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Sarepta’s Eteplirsen Walks Closer to Success

Posted: Published on April 5th, 2013

Eteplirsen keeps up its steady walk toward approval. Sarepta Therapeutics (NASDAQ: SRPT) reported the latest clinical study results today for its Duchenne muscular dystrophy, or DMD, drug. Shares in the biotech are up nearly 5% as the trading day winds down. Latest resultsSarepta's news related to results from 74 weeks into a phase 2b study of eteplirsen. Patients taking the drug demonstrated continued stabilization of walking ability on the six-minute walk test, a standard assessment of walking capability and clinical function for neuromuscular diseases. As with earlier points in this study, DMD patients taking eteplirsen showed significant improvement in walking ability compared to patients on placebo. This was true for groups taking the 30 mg/kg and 50 mg/kg dosages of the drug. Eteplirsen also continued to be well-tolerated. Sarepta reported that no patients experienced significant treatment-related adverse events or any serious adverse events. At week 62, one patient had a temporary elevation of urine protein, but that was resolved and the patient stayed in the study with no further problems. Chris Garabedian, Sarepta's president and CEO, said that the company was "encouraged to see a continued stabilization of walking ability in patients treated with eteplirsen for nearly one and a … Continue reading

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Atitude na Terapia para o Autismo The Son Rise Program® – Falsa Esperança é Coisa que Não Existe – Video

Posted: Published on April 5th, 2013

Atitude na Terapia para o Autismo The Son Rise Program - Falsa Esperana Coisa que No Existe By: Vencer Autismo … Continue reading

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Preventing Autism | What the Parents Say – Video

Posted: Published on April 5th, 2013

Preventing Autism | What the Parents Say We asked 1287 parents of children with autism, if asked how to reduce the rising rates of AUTISM, what would you say? Their answers speak to everyone who is... By: Regarding Caroline … Continue reading

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National Autism Network Launches Website with the Most Comprehensive and Up to Date Resources for the Autism Community

Posted: Published on April 5th, 2013

The National Autism Network organizes the Internet as it pertains to autism spectrum disorders providing both families and providers with a single source to get the latest information. The site includes the largest collection of autism related articles, hundreds of pages of information on diagnostics, early intervention, treatment models, education, evidence-based practice, and legislation along with discussion forums allowing parents and professionals to connect on an unlimited number of subjects, online seminars/webinars on various subjects and provider directory with over 34,000 providers. Cary, NC (PRWEB) April 05, 2013 When a child is diagnosed with autism, families spend an enormous amount of time scanning the Internet for answers. We created this resource to limit that time with information from credible sources as well as providing a forum in which parents, providers and individuals on the spectrum could unite and share stories, support and victories in one location. Our mission is to strengthen the autism community; we are all in this together, said Cari DeCandia, Director of National Autism Network. Every individual and family member that registers with the National Autism Network will have the capability to create a profile, participate in discussion forums, share success stories, view local events in their … Continue reading

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Scientists hope turtle DNA could lead to treatment for hypothermia, stroke

Posted: Published on April 5th, 2013

VANCOUVER - Scientists have decoded the DNA of the painted turtle and hope that unravelling the mystery of the reptiles may lead to medical breakthroughs for humans. Painted turtles are the most abundant turtle in North America, with a northernmost range from Ontario west to British Columbia, where they are listed as endangered on the Pacific Coast. The shelled reptile, named for the bright yellow stripes that adorn its body, can freeze solid and return to life when thawed. Brad Shaffer of UCLA's Institute of the Environment and Sustainability and one of the lead researchers says the turtle can also hold its breath for up to four days at room temperature without suffering oxygen deprivation. Shaffer and his colleagues hope solving the DNA puzzle may one day lead to innovations in treating hypothermia, heart attacks or strokes. The DNA information is being shared with 59 scientists around the world, including three researchers at UBC, Carlton and the University of Toronto who will continue the study. More here: Scientists hope turtle DNA could lead to treatment for hypothermia, stroke … Continue reading

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In The Spotlight: InVivo Therapeutics

Posted: Published on April 5th, 2013

The first human trial to evaluate a biopolymer scaffold product for traumatic spinal cord injury is all set to be initiated in the next few months by InVivo Therapeutics Holdings Corp. (NVIV: Quote). The FDA approved the company's Investigational Device Exemption, seeking permission to begin human studies to test its biopolymer scaffolding for the treatment of acute spinal cord injury, today. The company plans to finalize the details of the study over the next month or so, and expects to have all data to the FDA by the end of 2014. The study is expected to occur over approximately 15 months. In non-human primate studies involving monkeys and rodents with spinal cord injury, treatment with InVivo's biopolymer scaffolding has demonstrated functional recovery. According to the National Spinal Cord Injury Statistical Center, the annual incidence of spinal cord injury (SCI), not including those who die at the scene of the accident, is about 40 cases per million population in the U. S. or approximately 12,000 new cases each year. Currently, there are no treatment options to successfully restore function following a spinal cord injury. InVivo's biopolymer scaffolding product has also received Humanitarian Use Device, or HUD, designation. Devices that receive HUD … Continue reading

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InVivo Therapeutics Receives Approval from FDA for First Human Trial Using Biomaterials for Traumatic Spinal Cord Injury

Posted: Published on April 5th, 2013

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- InVivo Therapeutics Holdings Corp. (NVIV), a developer of groundbreaking technologies for the treatment of spinal cord injuries (SCI) and other neurotrauma conditions, today announced that the U.S. Food and Drug Administration (FDA) has approved the Companys Investigational Device Exemption (IDE) to begin human studies to test its biopolymer scaffold product, a technology developed to treat patients with acute, traumatic SCI. With this approval, InVivo intends to commence a first-in-man clinical study in the next few months that will test safety and performance of its biopolymer scaffold in five patients. The Company expects the study to occur over approximately 15 months. There are currently no treatment options approved by the FDA, or in clinical trials, to intervene directly in the spinal cord following SCI. The trial will be conducted at multiple U.S. hospitals, and work to gain Institutional Review Board (IRB) approval at Massachusetts General Hospital in Boston is already underway. Its heartbreaking for all of us for it to take even a minute longer than necessary to begin human studies, and weve all heard of, or experienced, treatments that have proven to be unsafe, but when conducting a first-in-man study, it is imperative to take the time … Continue reading

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Coming to grips with Parkinson ’s disease

Posted: Published on April 5th, 2013

There is no one symptom, no one level of symptoms, no one treatment for Parkinsons disease. The disease is as varied and unique as the persons diagnosed with it. To promote education about Parkinsons disease (PD) and ongoing research into the disease, the Parkinsons Disease Foundation has declared April as Parkinsons Awareness Month. Two Bureau County men are telling their stories of being diagnosed with PD, their treatments, and their encouragement to other families facing the disease. Looking back on to nearly 10 years ago, Gerrit Stevenson of Princeton said he lost his sense of smell around 2006-07 but never once thought it could have anything to do with an early onset of Parkinsons disease. But hes since learned the loss of the sense of smell is often, but not always, associated with persons developing PD. The tremors in Stevensons hands didnt begin until several years after he lost his sense of smell. By 2009, the occasional shaking of his hand became more frequent, and in February 2010, he went to the Mayo Clinic. But because the disease was in its earliest stages, a diagnosis wasnt able to be made at that time. He continued seeking answers and went to … Continue reading

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Mount Sinai Experts Call for Research on Prevalence of Delayed Neurological Dysfunction After Head Injury

Posted: Published on April 5th, 2013

Newswise One of the most controversial topics in neurology today is the prevalence of serious permanent brain damage after traumatic brain injury (TBI). Long-term studies and a search for genetic risk factors are required in order to predict an individuals risk for serious permanent brain damage, according to a review article published by Sam Gandy, MD, PhD, from the Icahn School of Medicine at Mount Sinai in a special issue of Nature Reviews Neurology dedicated to TBI. About one percent of the population in the developed world has experienced TBI, which can cause serious long-term complications such as Alzheimers disease (AD) or chronic traumatic encephalopathy (CTE), which is marked by neuropsychiatric features such as dementia, Parkinsons disease, depression, and aggression. Patients may be normal for decades after the TBI event before they develop AD or CTE. Although first described in boxers in the 1920s, the association of CTE with battlefield exposure and sports, such as football and hockey, has only recently begun to attract public attention. Athletes such as David Duerson and Junior Seau have brought to light the need for preventive measures and early diagnosis of CTE, but it remains highly controversial because hard data are not available that … Continue reading

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Experts call for research on prevalence of delayed neurological dysfunction after head injury

Posted: Published on April 5th, 2013

Apr. 5, 2013 One of the most controversial topics in neurology today is the prevalence of serious permanent brain damage after traumatic brain injury (TBI). Long-term studies and a search for genetic risk factors are required in order to predict an individual's risk for serious permanent brain damage, according to a review article published by Sam Gandy, MD, PhD, from the Icahn School of Medicine at Mount Sinai in a special issue of Nature Reviews Neurology dedicated to TBI. About one percent of the population in the developed world has experienced TBI, which can cause serious long-term complications such as Alzheimer's disease (AD) or chronic traumatic encephalopathy (CTE), which is marked by neuropsychiatric features such as dementia, Parkinson's disease, depression, and aggression. Patients may be normal for decades after the TBI event before they develop AD or CTE. Although first described in boxers in the 1920s, the association of CTE with battlefield exposure and sports, such as football and hockey, has only recently begun to attract public attention. "Athletes such as David Duerson and Junior Seau have brought to light the need for preventive measures and early diagnosis of CTE, but it remains highly controversial because hard data are not … Continue reading

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