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The FDA Has Approved A New Drug That Helps Treat Duchenne … – Paste Magazine

Posted: Published on February 22nd, 2017

The U.S. Food and Drug Administration (FDA) has approved a drug meant to treat Duchenne Muscular Dystrophy. The drug, Emflaza (deflazacort), is available in both tablet and liquid form and can be used by patients ages five years and older. Duchenne Muscular Dystrophy is a rare genetic disorder that causes progressive muscle deterioration and weakness. Symptoms of the disorder typically appear between between the ages of 3 and 5affecting the patient for years to come. It is common for patients to die in their 20s or 30s. Emflaza is a corticosteroid that works to decrease inflammation and reduce the activity of the immune system, which thus improves muscle strength in patients. Though corticosteroids are used to treat Duchenne Muscular Dystrophy in foreign countries, the FDA was hesitant to approve the drug prior to extensive research and testing. The FDAs ultimate approval of emflaza was based on two studies showing that male patients who took the drug had improved muscle strength, giving them more years with the ability to walk. In one study, 196 male patients between the ages of five and 15 were tested. After 12 weeks, there were improvements, and muscles appeared to be stronger in patients that took … Continue reading →

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Gilead Just Paid $125 Million to Nab a Big Market Advantage – Fortune

Posted: Published on February 22nd, 2017

It's actually a pretty good deal.Courtesy of Gilead Sciences Biotech giant Gilead Sciences ( gild ) is paying big bucks for the ability to speed one of its experimental treatments to the market. The firm paid $125 million to Sarepta Therapeutics to get its hands on a "priority review voucher," which can be used to reduce the Food and Drug Administration's review period for a drug from 10 months to six months, thereby potentially giving the company a crucial first-to-market advantage. Click here to subscribe to Brainstorm Health Daily, our brand new newsletter about health innovations. Sarepta won the coveted review voucher last September after receiving an approval from the FDA for the first drug in the U.S. to treat the rare movement disorder Duchenne muscular dystrophy. The agency's decision was hotly-contested as the treatment, called Exondys 51, hasn't actually proven its efficacy and has faced a revolt from some insurance companies , which are refusing to cover it. Sarepta chief Ed Kaye says that the money raised from its review voucher sale will go into funding R&D for future muscular dystrophy treatments. "The sale of the PRV provides an important source of non-dilutive capital to support the rapid advancement … Continue reading →

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For Parkinson’s patients, a cutting-edge treatment – Asbury Park Press

Posted: Published on February 22nd, 2017

Delgados Boxing in Atlanta is home to numerous champions in the professional boxing world. Now, theyre helping seniors with Parkinsons take back their lives by being their own champions in and out of the ring. USA TODAY NETWORK Patient Paul Detlefsen speaks with Dr. Asif Bashir and physicians assistant Jacqueline Cristini at JFK Neuroscience Institute in Edison.(Photo: Mike Zebrick) The tremors started when Paul Detlefsen was in his 30s. At 36, he got the diagnosis: Parkinsons disease. It was tough news for the Woodbridge resident, who works with his hands as a machine mechanic. I had real bad tremors in my right arm, he said. I didnt want to leave the house. I kind of got depressed. Three years ago he opted to treat the symptom with deep brain stimulation (DBS), which uses surgically implanted electrodes to stimulate areas of the brain that control movement. DBS therapy, which has been around for two decades, had the desired effect. Now you couldnt even tell I have Parkinsons, the 43-year-old said. Cautionary tale of the heart: Mom survives cardiac scare Patient Paul Detlefsen speaks with physicians assistant Jacqueline Cristini at JFK Neuroscience Institute in Edison.(Photo: Mike Zebrick) The only real drawback was … Continue reading →

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Prevacus Announces the Addition of Key Individuals to its Sports Advisory Board – P&T Community

Posted: Published on February 22nd, 2017

Prevacus Announces the Addition of Key Individuals to its Sports Advisory Board P&T Community 22, 2017 (GLOBE NEWSWIRE) -- Prevacus, Inc., a leading biopharmaceutical company focused on developing treatments for concussion (mild traumatic brain injury (mTBI)) and other neurological disorders; today announced the addition of Steve Mariucci, ... and more » Follow this link: Prevacus Announces the Addition of Key Individuals to its Sports Advisory Board - P&T Community … Continue reading →

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Sussex MS Treatment Centre

Posted: Published on February 22nd, 2017

Workingtogetherto improve thehealthand well being of people with Multiple Sclerosis and other chronic neurologicalconditions. Our Centre started out over 25 years ago as people in Sussex with MS decided to get together and fundraise for treatment equipment, now were also helping people with other long-term health conditions such as Stroke, ME, and children with Cerebral Palsy and Autism to name but a few. We support each other in keeping control of our lives, countering isolation, and managing symptoms. You can come to the Centre and access a wide variety of drug free treatments, exercise classes and social opportunities to cater for differing needs. Our aims are to maintain flexibility and mobility, provide pain relief, and promote well-being and a positive attitude. We strive to keep our services affordable and we will never refuse you a treatment for financial reasons. We offer a variety of individual treatments, such as Hyperbaric Oxygen Therapy, Shiatsu, Massage, Acupuncture, Chiropody and Osteotherapy. We also have group activities such as Seated Exercise, Yoga, Pilates, Physio Gym, Tai Chi, Voice Work, Meditation and Fitness classes. There is a very popular weekly coffee morning, and we hold regular social events with live music or other entertainment. You can … Continue reading →

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Risky treatment can stop multiple sclerosis for years – BBC News

Posted: Published on February 22nd, 2017

BBC News Risky treatment can stop multiple sclerosis for years BBC News A multiple sclerosis treatment being tested in patients can stop the disease for at least five years, say doctors. The risky therapy involves wiping out the person's immune system with strong cancer drugs and then rebooting it with a stem cell ... MS treatment that 'resets' immune system may halt disease progression for at least 5 yearsScienceBlog.com (blog) Multiple sclerosis: Stem cell transplantation may halt disease progressionMedical News Today Stem Cell Transplants May Help Some With MSWebMD Telegraph.co.uk -Mirror.co.uk -Daily Mail -The JAMA Network all 29 news articles » The rest is here: Risky treatment can stop multiple sclerosis for years - BBC News … Continue reading →

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A New Peptide Vaccine for Multiple Sclerosis Succeeds in Phase IIa – Labiotech.eu (blog)

Posted: Published on February 22nd, 2017

Apitope is developing therapeutic peptides to induce suppression of autoimmune responses towards self-antigens. Their recent phase IIa trial has revealed promising results for the treatment of multiple sclerosis. Multiple sclerosis (MS) is a hot topic these days.While arecentlong-term study revealedthe potential of hematopoietic stem cell transplantation for the treatment of MS, this treatment strategy is associated withlife-threatening complications mainly due toimmune deficiency. A very different approachis based on reinstating immune balance without causing global immune suppression. Apitopes technologyuses specific peptides, which induce an antigen-specific expansion of regulatory T cells the key mediatorsof immune tolerance. These cells are able to suppress autoantigen-specific helper T cells, which produce proinflammatory cytokines and are responsible for immune pathology in diseases such as MS. With itstechnology Apitope is trying to tackle four major disease areas includingMS, Graves Disease, Hemophilia type A and Uveitis. In 2009, Apitope licensed its MS lead ATX-MS-1467 to Merck KGaAin orderto develop and commercialize the treatment. However, Apitope announced that it regained full rights of their product in October 2016. Damage of the myelin sheath by the immune system in patients with multiple sclerosis. It seems like this was not a bad deal for Apitope as the company has now announced … Continue reading →

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New Blood Test May Make ‘Off the Rack’ MS Treatments Closer to ‘Designer Drugs’ – Everyday Health (blog)

Posted: Published on February 22nd, 2017

By Trevis Gleason Since the mid-1990s, the diagnosing process for most people suspected of having multiple sclerosis (MS) has included a lumbar puncture (also called a spinal tap) to look for certain antibodies and proteins, and an MRI scan to look for lesions in the brain. Previous to that, immersion into a hot tub (to see if symptoms worsened with heat) and evoked response tests may have been used. In the future, its possible that a test for blood biomarkers could become a standard part of the diagnostic process for MS and it could also help to determine the best form of treatment for those diagnosed. Earlier this month, Australian researchers announcedthey had discovered blood biomarkers of multiple sclerosis after 12 years of research. The biomarkers not only identify the presence of MS but also help to determine a persons stage of MS progression. While a commercial blood test using this research is not yet available, one is currently being developed. AnAustralian radio broadcaston the biomarker discovery gave a good sense of how the researchers see such a blood test for MS panning out. The researchers hope this type of blood testing might also prove helpful for people withmotor neuron … Continue reading →

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Studies provide fuller picture of testosterone benefits, risks – KFGO

Posted: Published on February 22nd, 2017

Wednesday, February 22, 2017 7:03 a.m. CST By Andrew M. Seaman (Reuters Health) - For men with low levels of testosterone, hormone-containing gels may offer a mixed bag of benefits and risks, according to a series of five new studies. Researchers found that for older men, hormone replacement therapy with testosterone gel might improve bone health and help avoid anemia, but it wouldn't help with thinking skills. Two studies looking at testosterone use and heart health produced mixed results. The new studies substantially improve the information available on testosterone treatment, said Susan Ellenberg, who co-authored four of the new studies. "There are definitely benefits and potential risks," said Ellenberg, a professor of biostatistics in the Perelman School of Medicine at the University of Pennsylvania in Philadelphia. "Its not an overwhelming conclusion that every man over 65 should be on this or stay away from it." Testosterone levels tend to decline as men age. Doctors sometimes prescribe testosterone gel - known as androgen replacement therapy. Four of the new studies are the last in a group of seven known as the Testosterone Trials, which evaluated the use of androgen replacement therapy in men over 65 with low testosterone unrelated to any … Continue reading →

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Heart disease different in women, but still just as deadly – Ahwatukee Foothills News

Posted: Published on February 22nd, 2017

One interesting difference between men and women involves the cardiovascular system and the development of heart disease and subsequent symptoms. There is growing awareness that when a woman experiences symptoms of heart disease, these symptoms may differ from what is universally recognized as typical cardiac symptoms. This is because of the differences in the male and female cardiac physiology. Heart disease in men generally involves the larger blood vessels supplying heart muscle whereas in women, heart disease often involves the smaller vessels. For men, when the larger vessels become narrowed because of atherosclerosis (so called hardening of the arteries) and plaques of fatty deposits, blood flow to heart muscle decreases. This causes the crushing pressure-type of chest pain known as angina. On the other hand, in the female heart, the smaller vessels become blocked before the larger ones. So a womans angina is more likely to be vague and expressed as fatigue, nausea, and/or dizziness. A womans description of heart pain can frequently be less dramatic and therefore draw less attention and less urgency. When women seek care, their providers may be misled by the presenting symptoms and a diagnosis of heart disease may be missed. In fact a large … Continue reading →

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