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Researchers identify novel therapy to treat muscular dystrophy

Posted: Published on June 26th, 2013

June 25, 2013 Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model. The findings, published in Human Molecular Genetics, represent a paradigm shift for the treatment of muscular dystrophy as well as a host of other disabling and devastating muscle diseases. The study was led by Mahasweta Girgenrath, PhD, assistant professor and director of the Muscle Disorders and Regenerative Biology Laboratory at BU Sargent College's Department of Health Sciences. Boston University (BU) researchers and postdoctoral fellows Jenny Yamauchi, Ajay Kumar, Lina Duarte, and Thomas Mehuron were collaborators on this study. Muscular Dystrophy type 1A (MDC1A) is the second most common form of congenital muscular dystrophy. Patients with this disease have poor muscle tone at birth, extremely compromised neuromuscular function, and are rarely able to walk independently. Most patients with MDC1A succumb to a premature death due to either respiratory complications or failure to thrive. Although significant strides have been made towards understanding the molecular and biochemical mechanisms underlying MDC1A, there remains no effective therapy in place to combat this lethal disease. The research team, led by Girgenrath, hypothesized that … Continue reading

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New Fycompa® (perampanel) Data Presented at International Epilepsy Congress (IEC)

Posted: Published on June 26th, 2013

New data provides additional evidence for use of Fycompa in partial-onset epilepsy HATFIELD, England, June 26, 2013 /CNW/ - New data from 11 abstracts, including two oral presentations, presented at the 30th International Epilepsy Congress (IEC) in Montreal, Canada provide additional data on the safety, efficacy and impact on quality of life (QOL) of once daily Fycompa (perampanel) as adjunct treatment in partial-onset epilepsy, the most common form of seizures. One oral presentation highlighted the low discontinuation rates seen with long term perampanel treatment amongst patients who could titrate to higher doses, and a second oral presentation showed that the reduction in seizures achieved with perampanel leads to significantly improved patient QOL, even after adjusting for treatment related side effects.[1],[2] The first oral presentation examined the long term retention rates and the reasons for discontinuation of treatment in over 1000 patients from Phase III trials who received adjunctive perampanel treatment for 24 weeks.[1]The results showed that the total discontinuation rates declined over time, from 7.9 % at 24-36 weeks to 2.0% at 72 weeks and this was mirrored by a decline in rates of discontinuation due to adverse events (AEs) from 2.6% at 24-36 weeks to 0.8% at 72 weeks. … Continue reading

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First Study of Diazepam Nasal Spray in People with Epilepsy Shows Feasibility of Dosing During Seizure

Posted: Published on June 26th, 2013

ARDSLEY, N.Y.--(BUSINESS WIRE)-- Acorda Therapeutics, Inc. (ACOR) today announced results of the first clinical study to assess pharmacokinetics, safety and tolerability of Diazepam Nasal Spray in people with epilepsy. Study findings were presented on June 25 at the biennial International Congress of the International League Against Epilepsy (ILAE) and International Bureau for Epilepsy (IBE), being held in Montreal, Canada. Diazepam Nasal Spray is being developed for the treatment of people with epilepsy who experience cluster seizures, also known as acute repetitive seizures. The study results showed that the Diazepam Nasal Spray pharmacokinetics are comparable whether it is administered during or immediately following a seizure. For people with epilepsy who experience cluster seizures, it is critical that treatment be administered as soon as a cluster is recognized, to prevent additional seizure activity, said Enrique Carrazana, M.D., Acordas Chief Medical Officer. Diazepam Nasal Spray offers a therapeutic alternative that can be administered rapidly and conveniently. The study was an open-label, multi-center clinical trial, comprising 31 people with epilepsy who were admitted to epilepsy monitoring units. Each received a single dose of diazepam, administered as one spray in each nostril, during or immediately following a seizure. Blood levels of diazepam were measured serially … Continue reading

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Autism experts will give a free lecture Friday at OHSU

Posted: Published on June 26th, 2013

Two of the nation's leading autism experts will offer a free public lecture Friday at Oregon Health & Science University. David G. Amaral and Sally J. Rogers of the University of California, Davis, MIND Institute plan to speak on new advances in research and treatment. They'll explain research on the role of prenatal autoimmune factors that lead to abnormal brain development in children who develop autism. And they'll describe early intervention programs that work in young children and may help resume normal developmental trajectories, according to an OHSU news release. Amaral studies the brains of children with autism spectrum disorders and coordinates a multidisciplinary analysis of children with autism, the Autism Phenome Project, aiming to define biomedical characteristics of different types of autism that might lead to more effective treatments. Rogers works with children with developmental disabilities and their families, especially young children with autism. She specializes in early intervention and developing treatment and educational interventions for persons with autism of all ages. Their talk starts at 5 p.m. in the OHSU Auditorium, 3181 S.W. Sam Jackson Park Road. - Katy Muldoon Here is the original post: Autism experts will give a free lecture Friday at OHSU … Continue reading

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TheSpeedGamers Play “Final Fantasy” in Week-Long Marathon to Help Kids with Autism, June 24-July 1, 2013

Posted: Published on June 26th, 2013

Arlington, TX (PRWEB) June 25, 2013 TheSpeedGamers kick off their Final Fantasy week-long video game marathon to help increase autism awareness and funds to benefit ACT Today! (Autism Care and Treatment Today!). The Final Fantasy marathon is held from June 24th through July 1st and can be watched live at http://www.thespeedgamers.com. The gamers have raised over $173,000 for ACT Today! over the past four years, and plan to dedicate this entire week to nonstop gaming with the goal of raising an additional $15,000 in support of the national non-profit. ACT Today!s mission is to provide resources and support for children with autism whose families cannot afford or access the necessary tools their children need to reach their highest potential. This includes therapy, medical care, social skills programs, assistance dogs, assistive technology, basic safety equipment, tuition for special needs schools, and assistance to military families with children with autism. "We really can't praise these young gamers enough," says ACT Today!'s executive director Nancy Alspaugh-Jackson. "This is the fifth marathon that TheSpeedGamers have hosted for our non-profit. We are truly grateful for such a dedicated and generous group of gamers who are joining us in the mission to help children with autism." … Continue reading

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Florida Autism Center Celebrates Success as Five Children with Autism Exit Treatment for "Mainstream" Classrooms

Posted: Published on June 26th, 2013

Tallahassee, FL (PRWEB) June 26, 2013 The Florida Autism Center (FAC) has a lot to celebrate. Five current clients, ages 2 - 8, exit FAC's program this summer. "Most businesses aren't thrilled to 'lose' clients," says Chrystin Bullock, FAC Executive Director, "but we are ecstatic when a little one leaves us because the therapy has been so good they stop needing it." Bullock goes on to say that she feels like sometimes people don't believe "recovery" from an autism spectrum disorder is possible. "Some people seem to see it as this mythical thing, like 'can that really be done?' But it definitely can," she states. In fact, every year FAC has a handful of clients exit treatment for mainstream preschool or kindergarten. The secret sauce of Florida Autism Center's treatment is Applied Behavior Analysis therapy implemented in a style all their own. The agency has an extreme focus on teaching students with autism meaningful social interactions, language, and play. A general course of treatment for a very young child diagnosed with autism is 18 - 36 months. Bullock says many clients can recover to the point of having skills and talents that are indistinguishable from those of same age peers … Continue reading

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Autism Speaks funds nearly $700,000 for high priority research

Posted: Published on June 26th, 2013

Public release date: 26-Jun-2013 [ | E-mail | Share ] Contact: Jane E. Rubinstein jrubinstein@rubenstein.com 212-843-8287 Autism Speaks New York, N.Y. (June 26, 2013) Autism Speaks, the world's leading autism science and advocacy organization, today announced awards totaling $685,968 funding new research in areas of high priority that will advance understanding and treatment of autism spectrum disorders (ASD). The new funding includes six translational postdoctoral fellowships and a study on dementia in older persons with autism. To date, Autism Speaks has committed nearly $199 million for research projects that advance understanding of the causes, prevention treatment and cure of ASD. While there has been unprecedented progress in the field of autism research, there is still much to understand about ASD. The rising prevalence, from the CDC prevalence estimate of one in 88 children to the most recent survey finding that one in 50 school-age children are diagnosed with ASD clearly establishes the urgency for progress in understanding the causes and finding effective therapies and treatments and underlies the need for research. Through research from the field's brightest postdoctoral scientists as part of the translational postdoctoral fellowship program, Autism Speaks seeks to accelerate the pace at which basic scientific discoveries are … Continue reading

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Center for Autism and Related Disorders Opens State-of-the-Art Treatment Center In Thousand Oaks, California

Posted: Published on June 26th, 2013

Thousand Oaks, CA (PRWEB) June 26, 2013 Center for Autism and Related Disorders, Inc. (CARD), one of the worlds most effective autism treatment centers which uses the evidence-based principles of applied behavior analysis, will host the opening of its Thousand Oaks treatment center on July 31, 2013, from 5:00 pm to 7:30 pm, at 325 E. Hillcrest Dr., Suite 140. The community is invited to tour the facility, meet the staff, and explore the cutting-edge CARD curriculum. Refreshments and child care will be provided. Special guests include 10-year-old Ethan Gorin, who received treatment from CARD and has recovered from autism, and world-renowned autism expert/clinical psychologist and CARD founder/CEO Doreen Granpeesheh, PhD, BCBA-D. Gorin will share his personal journey through autism. Dr. Granpeesheh will discuss CARDs mission and her dedication to helping individuals with autism lead healthy, productive lives. We are excited to be able to open the doors to effective autism treatment in Thousand Oaks, says Tasia Wells, M.A., CARD clinical manager designee. CARD has a team of highly-trained and experienced staff that focuses on evidence-based treatment. We work to provide individualized treatment plans for every child, so each child can see measurable results and, in some cases, recover from … Continue reading

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Common meds containing caffeine may be linked to stroke

Posted: Published on June 26th, 2013

Taking medications containing caffeine was tied to a doubled or even tripled risk of having a stroke in a new Korean study that might seem to contradict recent evidence suggesting coffee and tea exert protective effects. But the results may be in fact be in line with that research, according to the study authors, who point out that people who drank the least coffee were most at risk when taking caffeinated drugs. The products included mostly over the counter pain relievers, cold medicines and alertness aids containing small amounts of caffeine. "Caffeine is a vasoconstrictor, causing blood vessels to tighten and increasing the pressure of the blood flow," Nam-Kyong Choi of Seoul National University College of Medicine, who co-led the study, said in an email That effect on blood pressure could explain the possible link to strokes, but the study didn't investigate the mechanism, Choi said. The researchers selected 940 adult patients who had suffered a hemmorhagic stroke, wherein a blood vessel in the brain bursts and bleeds heavily. They then compared those patients to a group of similar people who had not suffered a stroke but had been hospitalized, and to a third group who had neither suffered a … Continue reading

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New nerve regeneration technique restores bladder function in paralyzed mice

Posted: Published on June 26th, 2013

Researchers have discovered a way to regenerate nerves in mice that have been damaged by spinal cord injuries, restoring their bladder function, Medical Daily reported. When paralysis occurs, the bladder is one of the organs most affected. Bladder control is maintained through signaling that occurs between the brain and the spinal cord. But after a spinal injury, that messaging is disrupted, often putting patients at a greater risk for urinary tract infections and kidney stones. Previous research has been unsuccessful at achieving any significant nerve regeneration in mice with spinal injuries. However, in the study published in The Journal of Neuroscience, researchers successfully achieved nerve regrowth by adding a combination of a previously inhibited enzyme, chonrdoitinase, and a fibroblast growth factor (FGF) to the site of the injury. After adding a graft to the site, the nerves in the spinal cord grew back by up to 12 millimeters, Medical Daily reported. Though the mice were not able to walk, they did experience a restoration of bladder function. Mice were 40 percent more capable of emptying their bladders completely and were able to willfully control their bladders. The treated mice could also hold nearly three times the volume in their bladders … Continue reading

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