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Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne …

Posted: Published on November 28th, 2012

CAMBRIDGE, MA--(Marketwire - Nov 26, 2012) - Sarepta Therapeutics ( NASDAQ : SRPT ), a developer of innovative RNA-based therapeutics, announced today a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in support of Sarepta's broad-based program for the treatment of Duchenne muscular dystrophy (DMD).Sarepta's collaboration is with University College London's (UCL) scientist, Professor Francesco Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the EU and US.The EU Health Innovation-1 2012 Collaborative research grant will support certain IND-enabling activities and clinical proof of concept studies for an exon 53-skipping therapeutic.Sarepta recently announced positive results from its extension study of its Phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD.Sarepta is also developing other PMO-based exon-skipping drug candidates for exons 45 and 50. "The recent compelling clinical data on eteplirsen targeting exon 51, which started with our work on the Phase I study in the UK, provides a strong foundation for using Sarepta's technology against exon 53," said Francesco Muntoni, professor of pediatric neurology and head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London. … Continue reading

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Blog : Sarepta’s DMD strategy – develop 20 similar drugs

Posted: Published on November 28th, 2012

Tuesday, November 27, 2012 Finding a treatment for Duchenne muscular dystrophy (DMD) wont just mean developing one drug, says Chris Garabedian, president and CEO of Sarepta Therapeutics (Nasdaq:SRPT). It will mean developing as many as 20. The fatal inherited disorder affects one in 3,500 young boys, confining most of them to wheelchairs by the age of 12. Few patients with it live past the age of 30. Years of telethons by the Muscular Dystrophy Association and comedian Jerry Lewis have raised awareness of the disease, but today, available treatments are mostly limited to steroids - which only prolong the boys ability to walk by 2-5 years - and surgery, including heart transplants, according to the National Human Genome Institute. After Sareptas announcement this week that it will begin development of a fourth drug candidate for the treatment of DMD, Garabedian told Mass High Tech that the company is close to understanding the very origins of the disease in mutations of one of the longest genes in the human body, which is responsible for creating the muscle protein, dystrophin. Seraptas approach is to develop drugs which skip certain sections of the DNA where mutations inhibit production of dystrophin. While that wouldnt … Continue reading

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Protein injection points to muscular dystrophy treatment

Posted: Published on November 28th, 2012

Public release date: 27-Nov-2012 [ | E-mail | Share ] Contact: Paddy Moore padmoore@ohri.ca 613-737-8899 x73687 Ottawa Hospital Research Institute November 27, 2012 Ottawa Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics. These results were published today in the Proceedings of the National Academy of Sciences by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults. "This is an unprecedented and dramatic restoration in muscle strength," says Dr. Rudnicki, a senior scientist and director for the Regenerative Medicine Program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute. He is also a Canada Research Chair in Molecular Genetics and professor in the Faculty of Medicine at the University of Ottawa. "We know from our previous work that this protein, called Wnt7a, promotes the growth and repair of healthy muscle tissue. In this study we show the same types of improvement in a mouse model of Duchenne muscular dystrophy. We found that Wnt7a injections increased muscle strength almost two-fold, … Continue reading

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Neuroscientist From French National Center for Scientific Research to Receive American Epilepsy Society 2012 Award for …

Posted: Published on November 28th, 2012

Newswise WEST HARTFORD, Conn., November 27, 2012 Richard Miles, Ph.D., Research Director, French National Center for Scientific Research, and Group Leader, Cortex and Epilepsy, Institute for the Brain and Spinal Cord, Centre Hospitalier Universtaire Pitie-Salpetriere, Paris, has been named recipient of the prestigious 2012 Epilepsy Research Recognition Award for Basic Science conferred by the American Epilepsy Society (AES). The award recognizes Dr. Miles for his highly original contributions in the understanding of electrical activity in neurons of the hippocampus, a brain structure associated with temporal lobe epilepsy. The Epilepsy Research Recognition Award is part of the AES pubic recognition program to encourage and reward basic and clinical investigators whose research contributes importantly to the understanding and conquest of epilepsy. This years award in basic science will be presented on December 1st during the Societys 66th annual meeting and scientific conference in San Diego, USA. Research by Dr. Miles and his collaborators has provided remarkable insight into the cellular connectivity or wiring of the human cortex. Among his many seminal discoveries is the observation that a single neuron can initiate a spontaneous electrical burst in a population or group of neurons, synchronous activity that could explain how seizures begin. His work … Continue reading

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Pingree School presents debut screening of a documentary on epilepsy

Posted: Published on November 28th, 2012

DOCUMENTING EPILEPSY: Hamilton native Louis Stanislaw grew up with epilepsy but kept it a secret from friends and teachers. Now a filmmaker, he explores the effect the complex disorder has on sufferers and their families in the documentary On the Edge: Living with Epilepsy. The films debut screening is at the Pingree School in Hamilton on Dec. 11 at 7:30 p.m. According to Stanislaws website, http://www.epilepsyontheedge.com, epilepsy affects 3 million people directly in the United States, and millions more through family members, friends, co-workers, and caregivers. Still, it is widely misunderstood. Life with epilepsy can be unpredictable, Stanislaw said. Medicines that control seizures can have staggering side effects. Seizures can be frightening and dangerous. They can be mistaken for aggression, with tragic results. Reactions and improper treatment can have devastating consequences. In a life with daunting hurdles, ignorance is usually the biggest one for people with epilepsy. On the Edge told through Stanislaws own experience and the stories of other epilepsy patients, their families, and health care providers documents difficulties and misunderstandings at every turn: from family life to school and friends, to leaving home and starting a career and forming lasting bonds. It looks at the alienation, depression, and … Continue reading

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Emerging Data on USL261 (Intranasal Midazolam) to Be Presented at the 66th Annual Meeting of the American Epilepsy …

Posted: Published on November 28th, 2012

MAPLE GROVE, Minn., Nov. 26, 2012 /PRNewswire/ --Upsher-Smith Laboratories, Inc. (Upsher-Smith) a privately held, specialty pharmaceutical company committed to the development of new treatments for diseases of the central nervous system, is proud to sponsor emerging data on USL261, its novel formulation of intranasal midazolam, at the 66th Annual Meeting of the American Epilepsy Society (AES) in San Diego, CA, November 30 December 4, 2012. "Upsher-Smith is committed to working toward improving the lives of people living with epilepsy," said Pamela J. Davis, M.D., Sr. Director Medical Affairs, Medical Strategy, Upsher-Smith. "USL261 (midazolam) is being developed for the intranasal rescue treatment of seizures in patients who require control of intermittent bouts of increased seizure activity, often called seizure clusters. This is a condition where few treatment options presently exist, particularly for the outpatient setting." USL261 (midazolam) is a benzodiazepine in an investigational formulation that is delivered intranasally. It is intended to be administered by a caregiver in an outpatient setting for the rescue treatment of seizure clusters without active inhalation by the patient. Upsher-Smith plans to sponsor presentations for three investigational drug programs in epilepsy at the AES annual meeting. Following is a guide to Upsher-Smith-sponsored presentations during the meeting. … Continue reading

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Elsevier Launches New Open Access Journal – Epilepsy & Behavior Case Reports

Posted: Published on November 28th, 2012

NEW YORK, November 26, 2012 /PRNewswire/ -- Elsevier, a world-leading provider of scientific, technical and medical information products and services, announces the launch of Epilepsy & Behavior Case Reports a new, online-only, Open Access journal devoted to the rapid publication of case reports on the behavioral aspects of seizures and epilepsy. As a sister journal to Epilepsy & Behavior, case reports published in the new journal will benefit from wide exposure and cross-promotion of the two titles. Epilepsy & Behavior Case Reports presents original peer-reviewed case reports that feature novel observations, significantly add to previously-reported observations, or provide compelling illustrations of important clinical principles related to the care of patients who have epilepsy and associated behavioral aspects. Topics are drawn from a variety of fields, including clinical neurology, neurosurgery, neuropsychiatry, neuropsychology, neurophysiology, neuropharmacology, and neuroimaging. Steve Schachter, Professor at the Department of Neurology, Beth Israel Deaconess Medical Center, Boston, MA, USA and Editor-in-Chief of Epilepsy & Behavior Case Reports said of the launch, "Case reports are the beacons of the scientific frontier, drawing the attention of the scientific community to previously unknown, unobserved, or unaccepted phenomena and thereby influencing the course of scientific progress. This new launch will provide the … Continue reading

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Challenges In Managing Women With Epilepsy Discussed On Epilog.us

Posted: Published on November 28th, 2012

MAPLE GROVE, Minn., Nov. 27, 2012 /PRNewswire/ --Approximately 1.5 million women and girls in the United States have epilepsy, of which nearly 500,000 are women of childbearing age.1,2 Treating women with epilepsy, especially women of childbearing age, presents significant challenges to physicians. Epilog.us [http://www.epilog.us], Upsher-Smith Laboratories, Inc's [http://www.upsher-smith.com] epilepsy-focused educational website, discusses these challenges and provides professional insight by some of the epilepsy community's most respected physicians including Dr. Cynthia Harden, Chief of the Comprehensive Epilepsy Care Center, North Shore University Hospital and Long Island Jewish Medical Center, and Dr. Christopher Skidmore, Director of Neurology Residency/Assistant Professor of Neurology, at Jefferson Medical College of Thomas Jefferson University. To view the multimedia assets associated with this release, please visit: http://www.multivu.com/mnr/59306-upsher-smith-epilog-website-challenges-in-managing-women-with-epilepsy "There are several issues that need to be considered when providing treatment to women with epilepsy of childbearing age including: hormonal fluctuations and their effect on epilepsy; drug interactions between oral contraceptives and antiepileptic drugs; and pregnancy," said Dr. Cynthia Harden. "As clinicians, we need to understand these challenges and identify an appropriate treatment plan unique to each patient." Dr. Christopher Skidmore added: "When discussing epilepsy and pregnancy with my female patients, the first and most important thing we discuss is … Continue reading

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Autism Live Wednesday, November 21, 2012 – Video

Posted: Published on November 28th, 2012

Autism Live Wednesday, November 21, 2012 Like Autism Live on Facebook at facebook.com Today on Autism Live, Ask Dr. Doreen features Dr. Doreen Granpeesheh answering questions on what high functioning Autism means, how diet and health relate to Autism and more. During Let?s Talk Autism with Shannon and Nancy recent news linking fevers in pregnant mothers is discussed along with flu shots. Michael and Pross Bahinyoza visit to talk about their mission and success in bringing their nephew with Autism from Uganda with the help of Nancy Alspaugh Jackson and Autism Care and Treatment Today! Truly a show to thankful for! Autism Live is a production of the Center for Autism and Related Disorders (CARD), headquartered in Tarzana, California, and with offices throughout the United States and around the globe. For more information on therapy for autism and other related disorders, visit the CARD website at centerforautism.comFrom:AutismLiveViews:0 2ratingsTime:01:59:59More inEducation Read more: Autism Live Wednesday, November 21, 2012 - Video … Continue reading

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Dramatic rise in autism prevalence parallels research explosion

Posted: Published on November 28th, 2012

ScienceDaily (Nov. 26, 2012) This month's issue of the Archives of General Psychiatry features an editorial commentary by Autism Speaks Chief Science Officer Geraldine Dawson, Ph.D. In it, Dr. Dawson describes how the dramatic progress in autism research has paralleled increased recognition of autism's prevalence and financial impact. "This issue of the journal features three articles on autism," she writes in her editorial. "A decade ago, the journal published about the same number of autism articles per year." Dr. Dawson also notes that, while the funding for autism research has dramatically increased over the last decade, it hasn't kept pace with the increasing scale of the public health challenges posed by autism. Despite an increase in research and funding, "we yet to fully describe the causes of ASD or developed effective medical treatments for it," Dr. Dawson writes. "[This issue's] articles point to an urgent need for more research on prenatal and early postnatal brain development in autism, with a focus on how genes and environmental risk factors combine to increase risk for ASD." In one of the three articles, scientists report a three-fold increase in autism risk associated with exposure to traffic-related air pollution during pregnancy and the first … Continue reading

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