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Low back and neck pain; 6 months after stem cell therapy by Dr Harry Adelson – Video

Posted: Published on April 23rd, 2014

Low back and neck pain; 6 months after stem cell therapy by Dr Harry Adelson Low back and neck pain; 6 months after stem cell therapy by Dr Harry Adelson http://www.docereclinics.com. By: Harry Adelson, N.D. … Continue reading

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Orbsen Therapeutics to take part in 6m liver disease clinical trial

Posted: Published on April 23rd, 2014

The Orbsen Therapeutics team. The company has already worked on a number of FP7-funded cell therapy projects Orbsen Therapeutics, a spin-out from NUI Galways Regenerative Medicine Institute (Remedi) has announced plans to partner with the University of Birmingham in a 6 million project to fight liver disease. The stem cell research company, which was founded by Prof Frank Barry and Prof Tom OBrien in 2006, will collaborate with the university on an EU FP7-funded project known by as Merlin (MEsynchymal stem cells to Reduce Liver INflammation). The project, which is led by Prof Phil Newsome, will advance Orbsens proprietary cell therapy to a Phase 2a clinical trial. Orbsen, which was recently shortlisted for this years Irish Times InterTradeIreland Innovation awards, has developed and is patenting a unique method of isolating therapeutic stem cells from human tissue at class-leading levels of purity. The companys proprietary ORB1-MSC (Orbsen developed mesenchymal stromal cells) therapy is being developed for several diseases, including inflammatory disease of the lungs and liver, diabetes, cardiovascular disorders, joint disease, kidney injury, organ graft rejection and wound repair. The total research budget for the Merlin project is close to 6 million of which 1 million will go directly to Orbsen … Continue reading

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New hope for Duchenne muscular dystrophy drug

Posted: Published on April 23rd, 2014

Sarepta Therapeutics Inc's shares soared 64 percent after it said that the U.S. Food and Drug Administration indicated an alternate path to approval for the company's experimental muscle disorder drug. The news provides a fresh lease of life for Sarepta, whose plans for a marketing approval the regulator deemed "premature" late last year, citing insufficient trial data. The agency has now indicated that safety and efficacy data from studies without placebo groups could support an approval application, analysts said. The regulator had earlier said that the company would need to compare the drug, eteplirsen, with a placebo. Patients treated with the drug in the new trials can now be compared with historical data from other patients with Duchenne muscular dystrophy (DMD), analysts said. Sarepta plans to file for approval by the end of the year and expects to get marketing nod in the second half of 2015. "Currently we have 2018 as the year of launch for eteplirsen, but this timeline could shave as much as 2+ years off that projection," Cowen & Co analyst Edward Nash said in a note. Sarepta is developing eteplirsen as a treatment for DMD, a degenerative disorder that hampers muscle movement and affects one … Continue reading

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Sarepta Therapeutics To File NDA For Muscular Dystrophy Drug

Posted: Published on April 23rd, 2014

By Estel Grace Masangkay Sarepta Therapeutics, a company focused on developing RNA-based treatments, announced its intent to file a New Drug Application for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) by the end of 2014. Eteplisen is the companys lead exon-skipping drug candidate under development for the treatment of patients with DMD who have a genotype disposed to skipping of exon 51. The company said it reached the decision to file an NDA by the end of the year based on a guidance letter from the FDA. According to the agency, an NDA for eteplirsen could be filed under a potential Accelerated Approval pathway. The FDA also provided guidance on an open-label, historically controlled confirmatory study of the drug as well as initial guidance on a placebo-controlled study on one or more of the companys follow-on DMD drug candidates. Chris Garabedian, president and CEO of Sarepta Therapeutics, said, As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study. We also appreciate that the … Continue reading

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Hopes high for muscular dystrophy drug

Posted: Published on April 23rd, 2014

Published: Monday, April 21, 2014, 10:03p.m. A new drug designed to slow the progression of Duchenne muscular dystrophy, a devastating disease that overwhelmingly affects boys and typically leaves them needing wheelchairs by their teens and dead in their 20s, has received a long-awaited boost from the Food and Drug Administration. Massachusetts-based Sarepta Therapeutics said Monday that FDA officials detailed a potential path forward for the drug, eteplirsen, and indicated a willingness to consider it for accelerated approval. The news puts eteplirsen one step closer to becoming the first approved treatment for Duchenne, a disease that affects about one in 3,500 boys globally. This provides the opportunity to get the drug approved and in the hands of all the boys who can benefit from it sometime in 2015, Sarepta chief executive Chris Garabedian said. I think that's a huge win for the [Duchenne] community and for Sarepta. The move follows a tumultuous year in which the FDA initially appeared headed toward swift approval of eteplirsen based on promising results from a trial involving 12 young boys, whose muscular deterioration seemed virtually halted by the drug. But the agency hit the pause button in November, saying it would be premature for Sarepta … Continue reading

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Mom: Colo. marijuana laws benefiting daughter's condition

Posted: Published on April 23rd, 2014

Mother says Colo. marijuana laws benefiting daughter's condition A young girl from the Kansas City area is making big improvements after taking advantage of Colorados new marijuana laws, according to her mom. KMBC 9 News reported about Sydni Yunek in February. VIDEO Yunek, 10, moved to Colorado from Kansas City with her mom, Holli Brown, for a special form of cannabis oil called Charlottes Web.Sydnis seizures, sometimes 125 per hour, have reduced by about 75 percent since Sydni started taking Charlottes Web last year, according to Browns observation. "It seems to do something that the pharmaceuticals can't do," Brown said. Brown first heard of Charlottes Web from other parents giving the treatment to their kids. It is a low-THC version of cannabis. Parents and patient advocates claim it has no psychoactive side effects on kids, though there is a lack of significant controlled studies on the cannabis oil. The treatment is not yet legal in Missouri or Kansas. Thats why Brown and her daughter moved from Missouri to Colorado last year to try and stop Sydni's seizures. More: Mom: Colo. marijuana laws benefiting daughter's condition … Continue reading

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Scott administration wants more restrictions on marijuana proposal

Posted: Published on April 23rd, 2014

Gov. Rick Scott and his surgeon general are balking at a proposal headed to the House floor that would open the door for a strain of marijuana that doesn't get users high but is believed to dramatically reduce seizures in children with a rare form of epilepsy. Instead, Scott wants a more limited approach that would put children with "intractable" epilepsy, as well as children and adults with other diseases, into clinical trials for the drug. The trials would require cooperation with the U.S. Food and Drug Administration or drug companies. A plan approved by the House Judiciary Committee on Monday includes language proposed by Scott that would create an "Office of Compassionate Use" within the Department of Health that would "enhance access to investigational new drugs for Florida patients through approved clinical treatment plans or studies." "Investigational new drug" studies are the first step in laboratory testing of drugs not yet approved by the Food and Drug Administration. Florida Surgeon General John Armstrong told the House committee Monday that he has concerns about other areas of the bill but that his approach is "patient-centered and provides access to medications of known content and dose" and could be operational within … Continue reading

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N.C. legislators pushing for insurers to cover more costs of autism treatment

Posted: Published on April 23rd, 2014

By Eric Garcia | Published 04/21/14 4:16pm A behaviorial treatment for students on the autism spectrum has been praised for its effectiveness but its not required to be covered by health insurance in North Carolina. Applied behavioral analysis is a form of therapy that helps people with autism reduce problematic behaviors and acquire skills such as language learning through the use of reinforcement. Legislators, educators and health officials hope to soon see a mandate that the treatment be covered by insurers. Applied behavioral analysis has a broad definition and is used in settings outside of psychological treatment, said Victoria Shea, a professor of psychology at UNC who works with TEACCH, a UNC-based service organization that works with people with autism. Its a way of applying learning principles and seeing the effect of that application, she said. And its effective, said Kristin Yonkers, clinical supervisor at the Mariposa School for Children with Autism in Cary. Its really the only approach that shows success time and time again, she said. A recent by the Centers for Disease Control and Prevention found that 1 in 68 U.S. children are on the autism spectrum. Last summer, the N.C. House of Representatives passed a bill … Continue reading

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Legislators urged to mandate autism coverage

Posted: Published on April 23rd, 2014

As parents detail their plight, insurers counter that services would cost too much POSTED: 03:47 a.m. HST, Apr 22, 2014 LAST UPDATED: 10:47 a.m. HST, Apr 22, 2014 For Gerilyn Pinnow, every dollar that goes to treat her son's autism is one less she's able to save for her daughter's college education. That choice is one reason Pinnow and other parents are pushing the Hawaii Legislature to pass a bill that would require insurance companies to cover treatments for autism, a move opposed by some insurers, who say it could lead to higher costs for people seeking coverage. The bill is named after Pinnow's 12-year-old son, Luke, who was diagnosed with autism at the Mayo Clinic in Minnesota. Traveling to get Luke diagnosed cost the family about $10,000, she said. Pinnow, a teacher who lives in Ewa Beach, said she had to tap into her retirement and short-sell her family's house to cover treatment costs. "No other family should have to go through that," she said in an interview Monday. Hawaii lawmakers continue to work out the details of Senate Bill 2054, which would require insurance companies to cover applied behavioral analysis and other treatment options. Applied behavioral analysis is … Continue reading

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Hawaii considers mandated autism care coverage

Posted: Published on April 23rd, 2014

HONOLULU (AP) For Gerilyn Pinnow, every dollar that goes to treat her son's autism is one less she's able to save for her daughter's college education. That choice is one reason Pinnow and other parents are pushing the Hawaii Legislature to pass a bill that would require insurance companies to cover treatments for autism, a move opposed by some insurers, who say it could lead to higher costs for people seeking coverage. The bill is named after Pinnow's 12-year-old son, Luke, who was diagnosed with autism at the Mayo Clinic in Minnesota. Traveling to get Luke diagnosed cost the family about $10,000, she said. Pinnow, a teacher who lives in Ewa Beach, said she had to tap into her retirement and short-sell her family's house to cover treatment costs. "No other family should have to go through that," she said in an interview Monday. Hawaii lawmakers continue to work out the details of Senate Bill 2054, which would require insurance companies to cover applied behavioral analysis and other treatment options. Applied behavioral analysis is widely viewed as the most effective treatment for autism spectrum disorders. But the Hawaii Medical Services Association, one of the state's largest insurance companies, opposed the … Continue reading

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