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Sarepta (SRPT) Targets Unmet Medical Need in the Treatment of Duchenne Muscular Dystrophy; Developing First Drug with …

Posted: Published on April 30th, 2013

67 WALL STREET, New York - April 29, 2013 - The Wall Street Transcript has just published its Biotechnology and Pharmaceuticals Report offering a timely review of the sector to serious investors and industry executives. This special feature contains expert industry commentary through in-depth interviews with public company CEOs and Equity Analysts. The full issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online. Topics covered: Health Care - Biotechnology and Pharmaceuticals - Executive Officer Interviews - Biotechnology and Pharmaceutical Investing - Orphan Drug and Biologics Manufacturing - Biotechnology and Pharmaceutical Companies Valuation Companies include: Gilead Sciences Inc. (GILD), Idenix Pharmaceuticals Inc. (IDIX), Achillion Pharmaceuticals, Inc (ACHN), Vertex Pharmaceuticals Incorpo (VRTX), Cubist Pharmaceuticals Inc. (CBST), Optimer Pharmaceuticals, Inc. (OPTR), Theravance Inc. (THRX), InterMune Inc. (ITMN), GlaxoSmithKline plc (GSK), Eli Lilly & Co. (LLY), Merck & Co. Inc. (MRK), Alexion Pharmaceuticals, Inc. (ALXN) and many more. In the following excerpt from the Biotechnology and Pharmaceuticals Report, an expert analyst discusses the outlook for the sector for investors: TWST: Which kinds of treatments or specific drugs do you think represent the most promising opportunities that investors may want to take a closer look at? Mr. Skorney: One of … Continue reading

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New molecule heralds hope for muscular dystrophy treatment

Posted: Published on April 30th, 2013

Javascript is currently disabled in your web browser. For full site functionality, it is necessary to enable Javascript. In order to enable it, please see these instructions. 6 hours ago by Liz Ahlberg Illinois chemists -- from left, undergraduate Kali A. Miller, graduate students Amin Haghighat Jahromi and Lien Nguyen, graduate student in Chemistry and professor Steven C. Zimmerman developed a small-molecule compound that could lead to therapeutic treatment for myotonic dystrophy, an as-yet untreatable disease. Credit: Ben Woloszyn (Phys.org) There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease. Steven C. Zimmerman, the Roger Adams Professor of Chemistry at the U. of I., led the group in developing and demonstrating the compound. The National Institutes of Health supported the work published in the journal ACS Chemical Biology. Myotonic dystrophy type 1 is the most common form of muscular dystrophy in adults, affecting one in 8,000 people in North America. It causes progressive weakness as the muscles deteriorate over time. … Continue reading

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Epilepsy Foundation To Present Epilepsy Therapy Project Lifetime Accelerator Award To Henrik Klitgaard, Ph.D …

Posted: Published on April 30th, 2013

LANDOVER, Md., April 30, 2013 /PRNewswire-USNewswire/ -- The Epilepsy Foundation announced today that Henrik Klitgaard, Ph.D., Vice President and Fellow, Neurosciences Therapeutic Area, UCB, has been named the recipient of the Epilepsy Therapy Project Lifetime Accelerator Award in recognition of his commitment and contributions to the field of epilepsy and to the people affected by it. Dr. Klitgaard will be honored at the Antiepileptic Drug and Device Trials (AED) XII Conference being held May 15-17, 2013, at the Turnberry Isle Miami Hotel, Aventura, FL. A leading and accomplished researcher in the epilepsy community, Dr. Klitgaard has conducted antiepileptic drug discovery in the pharmaceutical industry for more than two decades, most notably contributing to the discovery and development of levetiracetam. Currently, he serves as Vice President and Fellow, Neurosciences Therapeutic Area, UCB, where he has contributed to the research and development of multiple promising new anti-epilepsy drug candidates including PPSI, seletracetam and brivaracetam. Dr. Klitgaard was selected for the honor by an independent committee of global thought leaders and clinical investigators in epilepsy therapy discovery and development. "Dr. Klitgaard truly embodies the definition of the Lifetime Accelerator Award with his lifelong commitment to the epilepsy community and the advancement of important … Continue reading

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Autism stories: A support group in the page of a book

Posted: Published on April 30th, 2013

For parents of children with autism, comfort comes in talking with others who understand their struggles. A new book in the Chicken Soup for the Soul series speaks to families who are raising children "on the spectrum," with 101 stories from parents who have been there, including one by Christine Bakter of Haddonfield. In it, she describes a life-changing trip to the Barnegat Lighthouse with her two sons, both of whom have autism. The youngest, Ben (then 5 years old), flew up the stairs, exhilarated by the height and the view. Her oldest, Alex (then 8), was afraid of heights and didn't participate. The following summer, though, Bakter seized the opportunity of an early morning at the Cape Henry Lighthouse at Virginia Beach to work with Alex to overcome his fears. Coaxing him with the promise of a swim afterwards, they climbed the light together, despite Alex's strenuous opposition. Alex was thrilled to arrive at the top, and when it was over, he thanked her. She received "a very rare hug." Bakter calls the lighthouses her family has climbed more than 20 in the six years since beacons of hope. She sees them as symbols of her sons' triumphs over … Continue reading

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State of Autism Pt. 1: Tristan and Autumn One Year Later

Posted: Published on April 30th, 2013

There have been significant strides in autism treatment in West Virginia over the last year, but there is a long road ahead for autistic children and the services needed to treat them. Gov. Earl Ray Tomblin signed West Virginia's Autism Insurance Reform Law (HB 2693) April 2, 2012 at West Virginia University's Center for Excellence in Disabilities. It has a small Intensive Autism Service Delivery Clinic, which serves five children. Tristan and Autumn Hinebaugh are twins who receive hours of one on one instruction at the clinic. In 2012, their mother Tina described their behavior before they began treatment. "Tristan had two words at two and a half. He didn't know, if we were getting ready to go somewhere, what a coat was, how to put it on, nothing," Tina said. "Autumn would sit in a corner and play with a string." Now, the little boy who didn't even seem to notice there was a camera in the room can't keep his eyes or hands off of it while we record their session, and the little girl who seemed lost in her own world has found her words. "Chip," Autumn said, receiving praise and a Dorito from Board Certified Behavior … Continue reading

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State of Autism Part 1: Tristan and Autumn 1 Year Later

Posted: Published on April 30th, 2013

There have been significant strides in autism treatment in West Virginia over the last year, but there is a long road ahead for autistic children and the services needed to treat them. Gov. Earl Ray Tomblin signed West Virginia's Autism Insurance Reform Law (HB 2693) April 2, 2012 at West Virginia University's Center for Excellence in Disabilities. It has a small Intensive Autism Service Delivery Clinic, which serves five children. Tristan and Autumn Hinebaugh are twins who receive hours of one on one instruction at the clinic. In 2012, their mother Tina described their behavior before they began treatment. "Tristan had two words at two and a half. He didn't know, if we were getting ready to go somewhere, what a coat was, how to put it on, nothing," Tina said. "Autumn would sit in a corner and play with a string." Now, the little boy who didn't even seem to notice there was a camera in the room can't keep his eyes or hands off of it while we record their session, and the little girl who seemed lost in her own world has found her words. "Chip," Autumn said, receiving praise and a Dorito from Board Certified Behavior … Continue reading

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State workers' insurance to cover autism beginning July 1

Posted: Published on April 30th, 2013

By Joe Vardon The Columbus Dispatch Tuesday April 30, 2013 11:14 AM State workers and their families will receive insurance coverage for autism treatment beginning July 1 as a result of negotiations between the Kasich administration and labor unions. The two sides agreed to change contract language outlining employees health plans so that state workers and their families would be covered for physical, speech and occupational therapy, clinical therapeutic intervention, and mental and behavioral health outpatient services related to autism treatment, according to a news release. Late last year Kasich signed a directive to include autism services in Ohio's "essential health-benefit" package that federal law requires in every state beginning in 2014. He also said Ohio would make autism services available to state employees and their 40,000 covered children. This is another example of how State of Ohio labor and management representatives are working together and taking positive steps to impact the health outcomes for State of Ohio employees and their families, said Chris Mabe, president of the Ohio Civil Service Employees Association. For families that have autism in their lives, it can be a tough path, said Bob Blair, director for the states Department of Administrative Services. If they … Continue reading

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Autism Speaks, in partnership with Prometheus Research, deploys new online assessment portal for parents

Posted: Published on April 30th, 2013

NEW HAVEN, Conn., April 30, 2013 /PRNewswire/ -- Autism Speaks and Prometheus Research have teamed up to develop a new, more user-friendly assessment portal through which parents can complete surveys for use in autism research. The tool, called the Online Clinical System for Research (OSCR), allows parents to complete forms over the web and makes them accessible to Autism Speaks' Autism Genetic Resource Exchange (AGRE) scientists, among others. Assessments collected via OSCR provide important clues for autism research, increase the power of statistical analyses, and are easily shared with scientists and clinicians via a secure browser. The next step is to expand OSCR's impact by making it available to the Autism Speaks Autism Treatment Network. Prometheus Research, a leading provider of data management services for behavioral and biomedical research, developed the newest version of OSCR. "Our implementation placed a greater emphasis on usability but retained the functionality that researchers and parents value most," said Prometheus CEO, Dr. Leon Rozenblit. "This was a excellent use case for our new open-source electronic data capture tool, RexAcquire, part of the RexDB platform. RexDB simplifies data collection and, most importantly, automates the integration of survey data with internal study-specific information." Autism Speaks is now … Continue reading

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ARMC to hold stroke summit, screening event this weekend

Posted: Published on April 30th, 2013

ARMC Stroke Summit and Screening Event Where: ARMC, 400 N. Pepper Avenue, Colton. When: Friday: Stroke summit for health care professionals; Saturday: Stroke screening and assessment event for the public from 10 a.m. to 2 p.m. in ARMC Outpatient Care Center. Cost: Screening assessment event is free. (Only first 200 participants will be screened.) Information: ARMC, 909-580-1000, or https://www.arrowheadmedcenter.org/ COLTON -- Arrowhead Regional Medical Center is planning to hold a stroke summit for health care professionals on Friday and a stroke screening and assessment event for the public on Saturday, hospital officials announced Tuesday in a statement. The stroke summit is designed to provide health care personnel, such as physicians, surgeons, nurses and emergency medical services technicians, with updates on the latest standards of diagnosis and treatment, according to the statement. Guest speakers at the summit include the following ARMC doctors: Dan Miulli, new advancements in stroke-brain neurological rehabilitation; Robert Kounang, rehabilitation of the stroke patient; Kambiz Raoufi, importance of stroke core measures; Joachim Brown, importance of follow up with primary care physician; and Steven Fitzmorris, follow up of the stroke patient with atrial fibrillation. "As with many chronic diseases, the way we treat stroke and identify those who may … Continue reading

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Bouteflika stroke triggers Algerian crisis

Posted: Published on April 30th, 2013

The "minor stroke" that felled Algerian President Abdelaziz Bouteflika has plunged the government into crisis. ALGIERS, Algeria, April 29 (UPI) -- The "minor stroke" that felled Algerian President Abdelaziz Bouteflika has plunged the government into crisis as it is being pushed into the forefront of countering North Africa's jihadists, an enemy it's been fighting for two decades. The incapacitation of Bouteflika, 76, has heightened concerns about the stability of the energy-rich regional military heavyweight as the energy-rich country moves toward presidential elections next April amid a political power struggle. Bouteflika was flown to Paris Saturday night after suffering what the officials APS news agency termed a minor stroke. "His condition is not serious at all," said Algerian Prime Minister Abdelmalek Sellal, a close ally of Bouteflika. But Bouteflika's widely believed to have been in poor health for some time. He's rarely appeared in public in recent years and has repeatedly gone to France and Switzerland for medical treatment. He's dropped strong hints that he's ready to step aside for next year's election, that's likely to usher in a new era in Algerian politics. Bouteflika is one of the last of the generation of veterans who won the grueling 1954-62 war … Continue reading

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