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Sarepta Reports Significant Clinical Benefit With Eteplirsen In Phase IIb Trial

Posted: Published on July 25th, 2012

(RTTNews.com) - Sarepta Therapeutics (SRPT) announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test or 6MWT, over a placebo/delayed treatment cohort in a Phase IIb study in Duchenne muscular dystrophy or DMD patients. Eteplirsen administered once weekly at 50mg/kg over 36 weeks led to a 69.4 meter benefit compared to patients who received placebo for 24 weeks followed by 12 weeks of treatment with eteplirsen in the open-label extension. In the predefined prospective analysis of the study's intent-to-treat population on the primary clinical outcome measure, the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50mg/kg of the drug weekly showed a decline of 8.7 meters in distance walked from baseline, while patients who received placebo/delayed-eteplirsen treatment for 36 weeks showed a decline of 78.0 meters from baseline, for a statistically significant treatment benefit of 69.4 meters over 36 weeks. There was no statistically significant difference between the cohort of patients who received 30mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort. For comments and feedback: contact editorial@rttnews.com http://www.rttnews.com Read the original here: Sarepta Reports Significant Clinical Benefit With Eteplirsen In Phase IIb Trial … Continue reading

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Cure Duchenne Supported Sarepta Therapeutics Research Study for the Treatment of Duchenne Muscular Dystrophy

Posted: Published on July 25th, 2012

NEWPORT BEACH, Calif.--(BUSINESS WIRE)-- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by todays announcement by Sarepta Therapeutics (formerly AVI BioPharma) that its exon-skipping compound, eteplirsen, achieved a significant clinical benefit in a Phase IIb trial in Duchenne patients. CureDuchenne, along with Children National Medical Center in Washington, DC and the Foundation to Eradicate Duchenne, provided funding in 2010 for this research to progress into human clinical trials. This is a milestone for the company and patients alike. It demonstrated for the first time that eteplirsen achieved a highly significant clinical benefit on the 6-minute walk test, over a placebo/delayed treatment cohort in a Phase IIb trial in DMD patients. Eteplirsen was administered once weekly at 50mg/kg over 36 weeks, and patients on treatment demonstrated a 69.4 meter benefit (227 feet benefit) compared to those who received placebo/delayed treatment. The drug was well tolerated; there were no treatment-related adverse events, no serious adverse events and no discontinuations. The next milestone is at 48 weeks, and the data is expected in October. It will include analysis of dystrophin levels from muscle biopsies in addition to the clinical outcome measures. If … Continue reading

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Stroke patient reunites with lifesavers in Denver

Posted: Published on July 25th, 2012

Denver, CO(KSDK) - Thelast time Rick Hamilton was in Denver he'd suffered a stroke at 40,000 feet. Now he returns to the mile high city, reuniting with the team of doctors, nurses and paramedics who saved his life. When Rick Hamilton left Denver's University of Colorado Hospital, he was thankful to be alive. Now nine months later, in lieu of dozens of handwritten notes he'd thought about sending, Hamilton has stopped in to say thanks to the people who saved him. "I've been saying to myself all day, it's going to be tough to get through," said Hamilton. "But I think stroke causes that doesn't it." Hamilton a banking software salesman from Godfrey, Illinois was on a flight headed to Salt Lake City last October when he suffered a stroke at 40,000 feet. The planets seemed to align when a flight attendant noticed Hamilton's symptoms, an emergency room doctor on board diagnosed stroke, and the pilot landed the plane quickly. Hamilton ended up at UCH and in the hands of doctors, nurses and staff trained to deal with stroke and deal with it fast. "Rick as you probably all know was treated in 18 minutes," said Dan Meyers, UCH Communications … Continue reading

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Lazarus Effect ReCoverâ„¢ Thrombectomy Device Receives CE Mark

Posted: Published on July 25th, 2012

- Next-generation device for ischemic stroke treatment integrates gold standard stent-based clot retriever with integrated protective cover - CAMPBELL, California, July 24, 2012 /PRNewswire/ -- Lazarus Effect, a medical device company focused on improving treatment of acute ischemic stroke through effective removal of the blood clots that cause a stroke, announced today that the Lazarus ReCover Thrombectomy Device hasreceived CE Mark. The Lazarus ReCover is a next-generation ischemic stroke device that addresses a major limitation of stent-based clot retrieval, the current gold-standard technology for clot removal, by integrating a protective cover intended to prevent clot fragmentation or embolism and the potential for secondary stroke. Lazarus Effect plans to launch the Lazarus ReCover in Europe during Q1 2013. The company is currently evaluating distribution partners. Stent-based thrombectomy (clot removal) devices have demonstrated success "capturing" clots within the affected vessel, but often lose thrombus as the device is extracted. Among 124 Solitaire stent retriever cases evaluated during a recent European study, lost clot pieces traveled into an area of the brain previously not affected nine percent of the time, causing new areas of brain to experience stroke. The ReCover device comprises a nitinol wire-wrapped stent with a highly visible, radio-opaque core and … Continue reading

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Wonder pill could treat Alzheimer's, Parkinson's AND multiple sclerosis

Posted: Published on July 25th, 2012

A Phase I trial assessing the drug's safety in human patients is under way By Daily Mail Reporter PUBLISHED: 16:00 EST, 24 July 2012 | UPDATED: 19:32 EST, 24 July 2012 Early results from animal studies suggest new class of drug could be very effective against brain diseases One pill with the potential to treat conditions including Alzheimers disease, multiple sclerosis and strokes has been unveiled by scientists. Given early enough, it may even be able to stop full-blown Alzheimers from taking hold. It works by dampening down the inflammation thought to be at least partly to blame for many degenerative brain conditions, as well damage caused by head injuries and strokes. Animal tests have been encouraging and the pill has been given to humans for the first time, although the results have yet to be released. Early results from animal studies suggest it could be effective against a plethora of devastating brain conditions. They include Alzheimer's and Parkinson's disease, multiple sclerosis (MS), motor neurone disease, frontotemporal dementia, and complications from traumatic brain injury. See the original post here: Wonder pill could treat Alzheimer's, Parkinson's AND multiple sclerosis … Continue reading

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What's Up Doc: Cerebral palsy affects 3 per 1,000

Posted: Published on July 25th, 2012

Q: What exactly is cerebral palsy? A: Cerebral palsy (CP) is a general term that describes disorders of movement, muscle tone, gait and/or posture that are caused by a primary brain dysfunction prior to age three. CP is a non-progressive condition; the brain dysfunction does not typically get worse over time. It is the most common motor disability in young kids, affecting about 3 per 1000. The development of the human brain and nervous system begins just a couple of weeks after conception and continues for years after birth. CP can be caused by anything that causes the brain to develop abnormally; it is classified by the symptoms and limbs affected: - Spastic CP, accounting for over three quarters of cases of CP, manifests with stiff, tight, rigid and/or poorly stretchable muscles. It is called spastic hemiplegia if it affects one side of the body, spastic diplegia (paraplegia) if the legs are affected more than the arms, and spastic quadriplegia if all four limbs are affected. - Dyskinetic CP is manifested by abnormal body movements. - Ataxic CP is when walking coordination (gait) is abnormal. - Mixed CP is a mixture of any of these. The symptoms from CP range … Continue reading

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Dr. Jeff Hersh: Cerebral palsy affects 3 per every 1,000

Posted: Published on July 25th, 2012

Q: What exactly is cerebral palsy? A: Cerebral palsy is a general term that describes disorders of movement, muscle tone, gait and/or posture that are caused by a primary brain dysfunction prior to age 3. CP is a non-progressive condition; the brain dysfunction does not typically get worse over time. It is the most common motor disability in young kids, affecting about 3 per every 1,000. The development of the human brain and nervous system begins just a couple of weeks after conception and continues for years after birth. CP can be caused by anything that causes the brain to develop abnormally; it is classified by the symptoms and limbs affected: - Spastic CP, accounting for over three-quarters of cases of CP, manifests with stiff, tight, rigid and/or poorly stretchable muscles. It is called spastic hemiplegia if it affects one side of the body, spastic diplegia (paraplegia) if the legs are affected more than the arms, and spastic quadriplegia if all four limbs are affected. - Dyskinetic CP is manifested by abnormal body movements. - Ataxic CP is when walking coordination (gait) is abnormal. - Mixed CP is a mixture of any of these. The symptoms from CP range from … Continue reading

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Pill could treat brain conditions

Posted: Published on July 25th, 2012

A SINGLE pill has the potential to treat multiple brain conditions including Alzheimer's, Parkinson's and multiple sclerosis. Scientists have developed a new class of drug which can be taken orally and prevents the damaging effects of inflammation in the brain. Early results from animal studies suggest it could be effective against a plethora of devastating brain conditions. They include Alzheimer's and Parkinson's disease, multiple sclerosis (MS), motor neurone disease, frontotemporal dementia, and complications from traumatic brain injury. Two of the drugs, known as MW151 and MW189, have been patented by US scientists at Northwestern University in Chicago. They work by blocking excess production of damaging immune system signalling molecules called pro-inflammatory cytokines. New research published in the Journal of Neuroscience showed how early treatment with MW151 prevented the development of full-blown Alzheimer's in laboratory mice. Scientists say the drugs offer a completely different approach to treating the disease to others currently being tested. These target the accumulation of beta amyloid protein deposits in the brain which are a key feature of Alzheimer's. In contrast the new drugs are designed to stop inflammation disrupting wiring in the brain and killing neurons. See the rest here: Pill could treat brain conditions … Continue reading

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Northwestern study: 1 drug might fight Alzheimer’s, other brain conditions

Posted: Published on July 25th, 2012

By MONIFA J. THOMAS Health Reporter mjthomas@suntimes.com July 24, 2012 4:46PM 8-30-2010---General scenes of Northwestern University in Evanston---for affordable colleges story---The archway on Sheridan Road--on campus--Sun-Times photo by Tom Cruze storyidforme: 34011645 tmspicid: 12477858 fileheaderid: 5669370 Updated: July 24, 2012 9:37PM A new class of drug shows promise it might one day offer a new form of treatment for Alzheimers disease, as well as helping fight multiple sclerosis and traumatic brain injury, researchers from Northwestern University Feinberg School of Medicine and the University of Kentucky reported Tuesday. In tests on mice, the experimental drugs reduced inflammation in the brain. Such neuroinflammation is believed to play a key role in the progressive damage involved in Alzheimers and the other neurological conditions, as well as in stroke. Reporting in the Journal of Neuroscience, the researchers said they found that that one of the experimental drugs, known as MW151, significantly slowed the effects of Alzheimers disease in mice that were genetically engineered to develop the disease when given orally three times a week beginning at six months of age. The mouse study was designed to be comparable to when a human patient would begin to develop mild cognitive impairment, an early sign of … Continue reading

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New research determines how a single brain trauma may lead to Alzheimer's disease

Posted: Published on July 25th, 2012

Public release date: 24-Jul-2012 [ | E-mail | Share ] Contact: Jennifer Kritz 617-636-3707 Tufts University, Health Sciences Campus BOSTON (July 24, 2012, 5:00PM EST)A study, performed in mice and utilizing post-mortem samples of brains from patients with Alzheimer's disease, found that a single event of a moderate-to-severe traumatic brain injury (TBI) can disrupt proteins that regulate an enzyme associated with Alzheimer's. The paper, published in The Journal of Neuroscience, identifies the complex mechanisms that result in a rapid and robust post-injury elevation of the enzyme, BACE1, in the brain. These results may lead to the development of a drug treatment that targets this mechanism to slow the progression of Alzheimer's disease. "A moderate-to-severe TBI, or head trauma, is one of the strongest environmental risk factors for Alzheimer's disease. A serious TBI can lead to a dysfunction in the regulation of the enzyme BACE1. Elevations of this enzyme cause elevated levels of amyloid-beta, the key component of brain plaques associated with senility and Alzheimer's disease," said first author Kendall Walker, PhD, postdoctoral associate in the department of neuroscience at Tufts University School of Medicine (TUSM). Building on her previous work, neuroscientist Giuseppina Tesco, MD, PhD, of Tufts University School of … Continue reading

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