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FDA to grant breakthrough drug status to Glaxo’s drisapersen

Posted: Published on June 28th, 2013

PBR Staff Writer Published 28 June 2013 GlaxoSmithKline's investigational compound drisapersen is being granted Breakthrough Therapy status by the US Food and Drug Administration (FDA) for the potential treatment of patients suffering from Duchenne Muscular Dystrophy (DMD). The Breakthrough Therapy designation has been granted by the regulator, after vetting the results from the Phase II Study (DMD114117), submitted in April at Cold Spring Harbor. The phase II study reported that boys taking drisapersen were able to walk 35m more than those on placebo, with the difference maintained up to 48 weeks. Drisapersen, which will be used in the treatment of DMD caused by mutations in the dystrophin gene, is being developed by GlaxoSmithKline, licensed from Dutch company Prosensa Therapeutics. With an aim to expedite the development and review of drugs for serious or life-threatening conditions, the FDA created the Breakthrough Therapy designation and enacted it in 2012. Commenting on the FDA approval, CureDuchenne CEO and Founder Debra Miller said, "The Breakthrough Therapy designation means the FDA has reviewed the data for drisapersen and will provide additional resources." "These results validate our early efforts to fund novel research and offer hope for finding an effective and safe treatment for all those … Continue reading

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Statement from CureDuchenne Regarding Prosensa IPO

Posted: Published on June 28th, 2013

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne muscular dystrophy, congratulates Prosensa Therapeutics BV on its initial public offering. CureDuchenne has been a longtime supporter of the company, providing early-stage funding 10 years ago for Prosensas antisense (exon skipping) research that led to the development of drisapersen, a promising novel drug for Duchenne. The U.S. Food and Drug Administration recently granted Breakthrough Therapy designation to drisapersen. Drisapersen has shown promising results in Phase II clinical trials released in early 2013 and CureDuchenne looks forward to seeing Phase III data later this year. CureDuchenne was the first muscular dystrophy organization to recognize the potential of Prosensas exon skipping research and in 2004 committed $1.3 million to further that research. A few years later, venture capital firms validated CureDuchennes investment with several million dollars to continue their research. In 2009, GlaxoSmithKline committed up to $650 million to put four of Prosensas exon skipping compounds on the research and development path. CureDuchenne saw great potential in Prosensas leadership and research teams 10 years ago and is proud to have been an early supporter of the company, said Debra Miller, CEO and Founder, … Continue reading

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CureDuchenne Comments on U.S. Food and Drug Administration Breakthrough Therapy Designation for GlaxoSmithKline’s …

Posted: Published on June 28th, 2013

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne is pleased that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping compound for the potential treatment of patients with Duchenne Muscular Dystrophy. Drisapersen is being developed by GlaxoSmithKline plc (GSK) and licensed from Prosensa Therapeutics. The Breakthrough Therapy designation is one of several programs created by the FDA to expedite the development and review of drugs for serious or life-threatening conditions. It was enacted in 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). For more information on the Breakthrough Therapy designation, click here. The Breakthrough Therapy designation means the FDA has reviewed the data for drisapersen and will provide additional resources, said Debra Miller, CEO and Founder, CureDuchenne. This classification generally is for clinical programs that demonstrate medical significance. Duchenne is a disease that progresses very quickly and for which theres currently no cure, so on behalf of the Duchenne community, we are grateful to the FDA for recognizing the need and potential for this drug. We hope to see the drug approved soon. CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne, committed significant … Continue reading

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Epilepsy Foundation of Greater Los Angeles and Loma Linda University Medical Center Partner to Host Epilepsy Community …

Posted: Published on June 28th, 2013

Los Angeles, CA (PRWEB) June 28, 2013 The Epilepsy Foundation of Greater Los Angeles has partnered with Loma Linda University Medical Center to host the Epilepsy Community Forum at the Wong Kerlee Conference Center on the Loma Linda University campus on Sunday, June 30, 2013 in Loma Linda, CA. From 10:30 am to 3:00 pm, the Forum will cover a wide-array of topics pertinent to the epilepsy community. Parents of children may benefit from the presentation Growing with Epilepsy: Cognitive and Behavioral Disturbances as they work to respond to their childs needs. People of any age living with epilepsy will be interested in the topic on New Horizons in Treatment of Epilepsy: Update on Medications and Surgical Options. To delve further into treatments, the Forum will also highlight Dietary Treatments in Epilepsy. The inherent theme of the Forum empowering the community through education is most apparent with the closing session, when Ann Kinkor, the State Advocacy Coordinator for Epilepsy California, and Susan Pietsch-Escueta, the Executive Director of the Epilepsy Foundation of Greater Los Angeles, join together to present on Advocating in the School and Healthcare Arenas. With the exception of the closing session, all sessions are facilitated by physicians from … Continue reading

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Research and Markets: Epilepsy – Italian Drug Forecast and Market Analysis to 2022

Posted: Published on June 28th, 2013

DUBLIN--(BUSINESS WIRE)-- Research and Markets (http://www.researchandmarkets.com/research/gfd7gk/pharmapoint) has announced the addition of the "PharmaPoint: Epilepsy - Italy Drug Forecast and Market Analysis to 2022" report to their offering. Epilepsy is a brain disorder characterized by spontaneously occurring and recurrent seizures. The market is heavily driven by the sales of antiepileptic drugs (AEDs) targeted at seizure reduction. The AED market is currently dominated by UCB's Keppra and GlaxoSmithKline's Lamictal. Although both drugs have experienced significant generic erosion, they form the mainstay of epilepsy treatment in the nine markets and will continue to have significant market share during the forecast period. Other key drugs include older generation AEDs such as Pfizer's Dilantin, Abbott's Depakote, and Novartis' Tegretol and Trileptal which still have significant usage due to their longevity in the market. However, the AED dominance landscape will continue to shift towards newer generation drugs particularly following the recent market entry of GlaxoSmithKline's Trobalt/Potiga and Eisai's Fycompa which both offer first-in-class mechanisms of action. The main driver for growth in this market is the launch of new AEDs, the continued uptake of recent AEDs, and the lower-than-average rate of generic substitution in Italy compared with other countries, although this may change during the forecast … Continue reading

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Autism 204- Supporting Speech, Language and Communication with Mobile Technology REVISED – Video

Posted: Published on June 28th, 2013

Autism 204- Supporting Speech, Language and Communication with Mobile Technology REVISED Marci Revelli, a speech-language pathologist and augmentative/alternative communication specialist at Seattle Children's Hospital discusses how mobile techno... By: SeattleChildrens … Continue reading

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Autism Center Can Evaluate More Than 450 New Patients Annually, Up From 275

Posted: Published on June 28th, 2013

Newswise BUFFALO, N.Y. - For families with a child on the autism spectrum, early intervention is the mantra. The earlier their child is diagnosed, the sooner intervention can start, making a tremendous difference in the life of both the child and family. But early diagnosis depends on early evaluation and in too many cities, there just arent enough specialists to provide efficient diagnoses; most children show signs of autism spectrum disorder before they are three years old. In many places, it can take seven to nine months to get a diagnosis, explains Michelle Hartley-McAndrew, MD, clinical assistant professor of child neurology at the University at Buffalo and medical director of The Childrens Guild Foundation Autism Spectrum Disorder Center at Women & Children's Hospital of Buffalo. Seven to nine months is a lifetime for a parent to wait for a diagnosis with a child that young. Here at the center, we are working very hard to keep the waiting time down. A photograph of Hartley-McAndrew is at http://www.buffalo.edu/news/releases/2013/06/041.html. Now, thanks to the centers expanded schedule of clinics, which started in March, more Western New York families with children on the autism spectrum are receiving faster diagnoses. While each case varies, the … Continue reading

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Touro University helps families with autism

Posted: Published on June 28th, 2013

Touro University helps kids with autism and makes Las Vegas a better place to live Video by ktnv.com A doctor reminds people that when they see a child misbehaving in a store, that child could actually have a developmental disability tthat is causing their behavior Video by ktnv.com Henderson, NV (KTNV) -- More kids than ever are being diagnosed with Autism and these children and their families need treatment and help. They're finding it at Touro University's Center for Autism and Developmental Disabilities. "Very nice, what's your mom's phone number?" 12-year-old Steven has autism. For 3 years he's come for treatment to Touro University's Center for Autism and Developmental disabilities. And in that time, his family has seen dramatic improvements. Mom Chris Bailey says, "They've helped us transition him into middle school, issues that come up at home -- they're there for us. It's a family issue. It's not just the child it's everybody in the family and the center is there for everybody." Taneesha Porter is one of Steven's favorite therapists. But a variety of therapists -- with different specialties -- work together under the direction of Dr. Nicole Cavenagh. "Even if a child is only accessing 1 or … Continue reading

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Advocates seek insurance coverage for autism treatment

Posted: Published on June 28th, 2013

Raleigh, N.C. Advocates for people with autism want access to critical treatments, but legislation that would allow it is hung up in the General Assembly. Most insurance providers cover speech, occupational and physical therapy, but North Carolina is one of 17 states where insurance doesn't cover applied behavior analysis, also known as ABA therapy. The state House voted overwhelmingly last month to approve legislation that would require insurers to cover ABA therapy, but it has languished in a Senate committee since then. Scott Taylor said he and his wife paid $1,500 out of pocket for ABA therapy for their 11-year-old son, Daniel. He credits the strides his son has made to the therapy, which involves a team of therapists reinforcing positive behaviors and trying to eliminate negative ones. "It makes a big difference," Taylor said. "It is time intensive, one on one, and so it's not cheap." A year ago, he said, they had to halt the therapy because they could no longer afford it. "These children cannot wait on the treatment that they need and that their doctors are prescribing," said Lorri Unumb, vice president for government affairs for advocacy group Autism Speaks. "To have an insurance policy that … Continue reading

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Early brain stimulation may help stroke survivors recover language function

Posted: Published on June 28th, 2013

June 27, 2013 Non-invasive brain stimulation may help stroke survivors recover speech and language function, according to new research in the American Heart Association journal Stroke. Between 20 percent to 30 percent of stroke survivors have aphasia, a disorder that affects the ability to grasp language, read, write or speak. It's most often caused by strokes that occur in areas of the brain that control speech and language. "For decades, skilled speech and language therapy has been the only therapeutic option for stroke survivors with aphasia," said Alexander Thiel, M.D., study lead author and associate professor of neurology and neurosurgery at McGill University in Montreal, Quebec, Canada. "We are entering exciting times where we might be able in the near future to combine speech and language therapy with non-invasive brain stimulation earlier in the recovery. This could result in earlier and more efficient aphasia recovery and also have an economic impact." In the small study, researchers treated 24 stroke survivors with several types of aphasia at the rehabilitation hospital Rehanova and the Max-Planck-Institute for neurological research in Cologne, Germany. Thirteen received transcranial magnetic stimulation (TMS) and 11 got sham stimulation. The TMS device is a handheld magnetic coil that delivers … Continue reading

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