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Prosensa’s IPO and the Value of the BIO Investor Forum

Posted: Published on September 11th, 2013

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Our primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntingtons disease. We completed an IPO onto NASDAQ in June of this year. Prior to last years BIO Investor Forum in San Francisco, we were still in the very early stages of contemplating an IPO onto the U.S. markets. Our decision to list in the U.S. markets was not an easy one. Given a European biotech company had not accomplished its primary listing on NASDAQ in almost a decade, it was imperative that we gain exposure with U.S.-based investors prior to embarking on the road to an IPO. As much of the investment in the biotech industry is concentrated in the U.S., we felt that listing on NASDAQ would provide us with the necessary access to capital to be able to further develop our DMD portfolio of 6 unique compounds. BIOs legislative efforts leading to the passage of the JOBS Act in 2012 have been very supportive for IPOs. We evaluated several conferences that … Continue reading

Posted in Muscular Dystrophy Treatment | Comments Off on Prosensa’s IPO and the Value of the BIO Investor Forum

Eisai's EMEA Operation Becomes US Supply Centre for Epilepsy Drug Fycompa® (Perampanel)

Posted: Published on September 11th, 2013

HATFIELD, England, September 11, 2013 /PRNewswire/ -- FDA approval marks Eisai's continued investment in UK high tech drug manufacturing facility Japanese pharmaceutical company Eisai today announces that the US Food and Drug Administration (FDA) has approved the US supply of the epilepsy drug Fycompa (perampanel) from its EMEA (Europe, Middle East, Africa, Russia and Oceania) Knowledge Centre based in Hatfield, UK. The Hatfield site will manufacture six different strengths of tablet for export to the US. The FDA supply approval for perampanel is the latest in a series of achievements for Eisai in the UK. In addition to its existing EMEA sales and marketing, R&D, and manufacturing operations in Hatfield, Eisai recently announced the construction of new 2,900m2 facility for the global packaging of potent compounds which expands the current 7,800m2 Hatfield site by nearly 40%. The Hatfield site is the Eisai EMEA headquarters and is growing in significance to the company's worldwide business, as it becomes a global supply centre for innovative new products. Perampanel was jointly developed by Japan and the UK and, as a result, Eisai continues its commitment to a long-term global business structure and investment into further research and development in the UK. Gary Hendler, … Continue reading

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How Do I Use Skills®? – Video

Posted: Published on September 11th, 2013

How Do I Use Skills®? Like Autism Live on Facebook at http://facebook.com/autismlive Dr. Adel Najdowski answers a parent's question about how to use Skills®. Skills® is a comprehe... By: AutismLive … Continue reading

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Autism Therapy Chennai | Autism Treatment Centre India | Autism Success Stories Tamilnadu – Video

Posted: Published on September 11th, 2013

Autism Therapy Chennai | Autism Treatment Centre India | Autism Success Stories Tamilnadu http://www.autism-ent-specialist-chennai.com D.O.A.S.T. Integrated Therapy Centre for Autism Chennai, Tamil Nadu, is one of the best autism treatment center ... By: Doast Autism Center … Continue reading

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Enter the chamber: Frisco business sees results from Hyperbaric Oxygen Therapy

Posted: Published on September 11th, 2013

Several years ago Frisco resident Paul Mattson never would have imagined he could use oxygen to fuel his own therapeutic business. A biologist and a chemist by education, Mattson first began exploring the benefits of Hyperbaric Oxygen Therapy in 2008 when his son was diagnosed with autism. Shortly after he purchased his own hyperbaric chamber and opened Mountain Hyperbarics at 101 Main St. in Frisco. During the last five years, Mattson and Jeff Miller, a respiratory therapist and hyperbaric chamber technician, have performed hundreds of treatments, including more than 140 on Mattsons son, and theyre seeing marked improvements in their clients. But Hyperbaric Oxygen Therapy is still considered an investigative treatment, which means that although it is approved for certain conditions, and may even be covered by health insurance, doctors are still exploring its effectiveness in people with brain disorders, stroke victims or patients undergoing radiation and chemotherapy for cancer, among many other conditions. Even though it hasnt yet been proven as a beneficial treatment across a whole gamut of medical conditions, Hyperbaric Oxygen Therapy received national attention when it was used to treat soldiers returning from Iraq and Afghanistan with traumatic brain injuries caused by improvised explosive devices and … Continue reading

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Racial and ethnic differences seen in survival, care needs with certain strokes

Posted: Published on September 11th, 2013

WEDNESDAY, Sept. 11 (HealthDay News) -- Among patients with a certain type of bleeding stroke, racial and ethnic differences exist in their outcomes, a large new study finds. Researchers looked at data from more than 31,600 patients treated in U.S. hospitals for subarachnoid hemorrhage between 2005 and 2010. This type of stroke is caused by a ruptured aneurysm -- when a weak spot in one of the blood vessels supplying the brain breaks. The overall death rate among the patients in the study was 22 percent, and 42 percent of them required institutional care after they left the hospital. Compared to white patients, Asian/Pacific Islander patients were more likely to die in the hospital, while Hispanic patients were less likely to die, according to the study, which was published online Sept. 10 in the Journal of Neurosurgery. Black patients were more likely than whites to require institutional care after being discharged from the hospital, but their risk of death while hospitalized was similar to that of white patients, the University of Toronto researchers found. The likelihood of needing institutional care after leaving the hospital was similar among white, Hispanic, Asian/Pacific Islander and Native American patients. "[We] believe that there could … Continue reading

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Anti-inflammatory and anti-thrombogenic effects of atorvastatin in acute ischemic stroke

Posted: Published on September 11th, 2013

Public release date: 10-Sep-2013 [ | E-mail | Share ] Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research The population of northeastern China has a high incidence of ischemic stroke. Previous studies have shown that intracranial large-artery atherosclerosis is one of the main causes of ischemic stroke, and that the mechanisms are related to inflammation and thrombosis of the affected arteries. A recent study by Lianqiu Min and colleagues from Liaoning Medical University observed the effects of atorvastatin treatment in 89 patients from northeastern China with acute ischemic stroke caused by intracranial large-artery atherosclerosis by measuring changes in the levels of markers of inflammation, thrombogenesis, and hyperlipidemia. The researchers found that atorvastatin treatment decreased the levels of markers of inflammation, thrombogenesis, and hyperlipidemia, suggesting that atorvastatin reduces inflammation and thrombogenesis independent of its lipid-lowering effects in patients with acute ischemic stroke caused by large-artery atherosclerosis. Most previous studies of patients with acute ischemic stroke caused by large-artery atherosclerosis focused mainly on measurement of the level of a single marker such as C-reactive protein, fibrinogen, or D-dimer. The study, reported by Lianqiu Min and colleagues in the Neural Regeneration Research (Vol. 8, No. 23, 2013), measured all three of these values … Continue reading

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Treat heart attacks and strokes at home, says senior doctor

Posted: Published on September 11th, 2013

Sir Bruce insisted that patients would not be worried about being treated at home. People trust their GPs, people trust hospitals, but they also trust ambulance services, he said. They are very highly trained people. Theres a big opportunity there for us to see how we can use the expertise the ambulance services have. An NHS England spokesman confirmed that more home treatment was on the table in Sir Bruces review. Fundamental change was needed to meet burdens on A&E departments, he said. It was not clear last night exactly how the policy would work, but one option would be for paramedics to provide heart attack victims with anti-clotting drugs and stabilise them at home, before taking them to a specialist clinic to recover, thereby bypassing accident and emergency. Moving towards more home treatment for acute cases would reverse recent trends in medical practice. Over the past decade paramedics have provided less and less treatment to heart attack victims, and instead take patients directly to specialist heart attack centres where they undergo a procedure to open up a collapsed/blocked artery using a tiny tube. About 100,000 people suffer a heart attack each year, but last year 200 heart attack victims … Continue reading

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Drug free medical treatment

Posted: Published on September 11th, 2013

WEST HARTFORD, Conn. (WTNH) -- A drug free option for pain is now available in Connecticut. A nerve injury can be extremely painful. At times medication and other treatments can not bring relief. Now there's a new option that's non-invasive and drug free. Michelle Lyster tore a tendon in her right ankle, but after surgery she developed reflex sympathetic dystrophy. "Which causes severe burning pain, hypersensitivity," Lyster said. Doctors could do little for her excruciating pain. "I've been on a cane or crutches for the entire six years," Lyster said. Now a new electrical stimulation therapy offered at the Pain Treatment Center at Hartford Hospital is easing much of Lyster's pain. "The sensitivity is really down," she said. Dr. Jonathan Kost says it sends a no-pain message through electrodes. "Electrodes are essentially placed in the vicinity of the nerve injury," said Dr. Kost. "This new device resets the pain signal of how it's being sent eventually into the spinal cord and up to the brain as not being as painful as it was prior to the treatment." "It feels like a bee stinging," Lyster said, "but it's not painful and then it gets to a point where it's more of … Continue reading

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FOLD Cuts A Deal, SNTA On Watch, NanoString Makes Mega Leap, GALT Vaults

Posted: Published on September 11th, 2013

Amicus Therapeutics (FOLD: Quote) has entered into a collaboration with Biogen Idec (BIIB) to discover, develop and commercialize novel small molecules for the treatment of Parkinson's disease. The collaboration will build upon preclinical studies at Amicus and independent published research that suggest increasing activity of the lysosomal enzyme glucocerobrosidase (GCase) in the brain may correct alpha-synuclein pathology and other deficits associated with Parkinson's disease. FOLD closed Tuesday's trading at $2.66, up 4.31%. Conatus Pharmaceuticals Inc. (CNAT: Quote), which made its stock market debut as recently as July of this year, has initiated a phase II study of Emricasan in patients with severe alcoholic hepatitis. This study is being conducted by the Translational Research and Evolving Alcoholic Hepatitis Treatment (TREAT) Consortium consisting of the Mayo Clinic Rochester, Indiana University, and Virginia Commonwealth University in collaboration with the National Institute on Alcohol Abuse and Alcoholism. CNTA closed Tuesday's trading 0.47% higher at $8.62. Exelixis Inc. (EXEL: Quote) has initiated a phase III trial, dubbed CELESTIAL, comparing Cabozantinib with placebo in patients with advanced hepatocellular carcinoma who have previously been treated with Sorafenib. The primary endpoint for the trial is overall survival. EXEL touched a new 52-week high of $5.64 on Tuesday before … Continue reading

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