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FDA Fast Tracks Muscular Dystrophy Drug backed by Woodford – Labiotech.eu (blog)

Posted: Published on May 31st, 2017

The FDA has handed out a Fast Track Designation to AMO Pharma for its key asset AMO-02 to treat congenital myotonic dystrophy. The British-American biotech hybrid AMO Pharma is advancing asmall pipeline of drugs for rare genetic disorders. The biotech, which is backed by famous UK Investor Neil Woodford, is currently pushing its lead candidate AMO-02 through Phase II as the first-ever treatment for myotonic dystrophy. Now, AMO will also be backed by regulatory agencies, after the FDA today announced that it has granted Fast Track Designation for AMO-02. The Fast Track Designation paves the way for an accelerated approval process for AMOs lead drug and includes a priority review that clips four months off the FDAs decision process. This is great news for patients suffering from the disease, which represents the most common form of muscular dystrophy, and for which there is currently no approved therapy available. AMO-02 tries to tackle the most severe form of myogenic dystrophy known as congenital DM1. The drug works by inhibiting theglycogen synthase kinase 3 beta (GSK3), which is extra active in patients with DM1 as well as duchenne muscular dystrophy. So far, theinhibitor was able to demonstrate pre-clinical efficacy in transgenic models. … Continue reading

Posted in Muscular Dystrophy Treatment | Comments Off on FDA Fast Tracks Muscular Dystrophy Drug backed by Woodford – Labiotech.eu (blog)

CD Access to Provide Special Access Program for Duchenne … – Business Wire (press release)

Posted: Published on May 31st, 2017

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--CD Access is a new nonprofit that provides patients access to drugs and therapies that are currently unavailable through clinical trial or prescription. CD Access first program allows Duchenne muscular dystrophy patients in Canada that previously participated in a clinical trial for drisapersen to begin redosing through a Health Canada Special Access Programme. BioMarin transferred their supply of exon 51 skipping drug, drisapersen, to CD Access for distribution to boys who were participants in previous clinical trials in Canada. Former trial participants in Canada will need to contact their physician to confirm eligibility. The drug will be administered at Childrens Hospital of Western Ontario, Childrens Hospital of Eastern Ontario, Childrens & Womens Health Centre of BC and potentially one additional site in Canada. We are delighted to provide access to eligible patients who benefited from drisapersen, said Debra Miller, CEO and founder of CureDuchenne and the newly formed CD Access. We believe it is important for Duchenne patients who participated in the clinical trial to have access to investigational drugs when and where there are no approved options for treatments for this rare disease. When BioMarin discontinued development of drisapersen we received multiple calls from parents who … Continue reading

Posted in Muscular Dystrophy Treatment | Comments Off on CD Access to Provide Special Access Program for Duchenne … – Business Wire (press release)

Cape Town ‘teen mom’ uses daughter’s cerebral palsy to inspire … – News24

Posted: Published on May 31st, 2017

Cape Town When Nosphiwo Queeneth Gum gave birth to her daughter at 17, she could have hardly realised that one day she would be forced to say goodbye forever. And that she would emerge stronger, no longer frightened about death, and able to help other youths with difficult life choices. It was not long after little Simamkele was born in 2003 that doctors revealed she had Cerebral Palsy, a disorder that affects movement, motor skills and muscle tone. - Health24: Cerebral Palsy I thought: is that a boys name? They took her to hospital, did tests, and it was just a scary thing for me because I didnt know what was happening, the now 30-year-old said on Tuesday. She felt she had two choices either stressing about it or accepting it. I realised that after everything, as young as I was, she is my child and that comes from a place where I didnt really feel like I belonged anywhere and here I get given a responsibility. Gum is now a counsellor for Salesian Life Choices, a Cape Town-based social enterprise investing in youth. Campaign She was speaking as the organisation launched the 30 Stories in 30 Days campaign for … Continue reading

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Pro Dancer With Cerebral Palsy Calls Disability ‘Best Tool’ God Has … – CBN News

Posted: Published on May 31st, 2017

From a very young age, Jerron Herman had dreams of making it big in the cutthroat world of performing arts. Though he grew up in a small town in northern California, the self-described theater geek always knew hed end up in New York City. Herman was born with cerebral palsy, which by most peoples estimates would prevent him from fulfilling his ambitious goals. But in a recent interview with Faithwire, the now-professional dancer and choreographer shared how his Christian faith has enabled him to achieve the impossible. Herman, 26, initially came to New York with the goal of becoming a professional writer. Im a theater geek, and a writer first, he explained. I wanted to be a storyteller through theater, primarily. And I wanted to write for Broadway, and I wanted to write for television that was my dream. It wasnt until midway through college that Herman first considered a career in dancing. I dont think I ever really decided [that I wanted to be a dancer], he told Faithwire. It was really a series of invitations that led to a career. Photo credit: Andria May-Corsini The first invitation came from choreographerSean Curran, who runs his own dance company in … Continue reading

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Inspirational 10-year-old with cerebral palsy mixes with celebrities at glittering fundraising event – WalesOnline

Posted: Published on May 31st, 2017

An inspirational 10-year-old with cerebral palsy mixed with stars including Craig David, Fleur East and Paloma Faith at one of the most prestigious fundraising events in the UK. Mia Thorne, who has congenital hemiplegia, attended the Caudwell Childrens annual Butterfly Ball last week and mum Karene said by the end of the event celebrities were actually coming up to her asking for a photo. She was representing Wales as the child ambassador at the event and now counts celebrities such as Peter Andre, Blues Lee Ryan, and former Queen of the Jungle Vicky Pattison among her pals having met more than once. Mum Karene said: She was like a mini celebrity and spent lots of time chatting to all of the different stars. It was a truly amazing experience with all the celebrities walking the purple carpet. Kelvin Fletcher from Emmerdale and Lee Ryan were particularly amazing with Mia and danced with her. It was an eventful, unforgettable night and Mia absolutely loved every minute of it and having her photos taken by the national newspapers. I am very proud of her. Mia, from Pontypridd, and a pupil at Coedpenmaen Primary School, has congenital hemiplegia, a form of cerebral palsy … Continue reading

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Boy (6) with severe cerebral palsy awarded 778k over … – Irish Independent

Posted: Published on May 31st, 2017

The 778,606 settlement figure covers the care needs of Martin Gibson for the next year as well as past care provided by the boy's parents. Martin, through his mother Sarah Gibson, of Castle Manor, Newcastle, Co Wicklow, sued the NMH for alleged negligence over the circumstances of his birth on July 3, 2010. It was claimed there was an alleged failure to properly manage and monitor the labour, delivery and birth of the baby. There was an alleged failure, at a review of the labour, to recognise or respond to abnormalities present on a cardiotocography (CTG), including persistent late decelerations. There was also an alleged failure to recognise a pathological first stage CTG necessitating the taking of urgent action. It was also claimed there was as failure to proceed to an emergency caesarean section and there was an alleged inappropriate delay in the decision to deliver the baby by caesarean. Martin was flat on delivery and was later transferred to Our Lady's Hospital for Sick Children. The hospital denied the claims. The High Court recently found that the defendant was 70 per cent liable in the case. His counsel, Denis McCullough, said Martin is unable to communicate, uses a wheelchair … Continue reading

Posted in Cerebral Palsy | Comments Off on Boy (6) with severe cerebral palsy awarded 778k over … – Irish Independent

Chilhowee Shrine Club Rodeo is this weekend – The Daily Post-Athenian

Posted: Published on May 31st, 2017

The "greatest show on dirt" makes its return with the annual Chilhowee Shrine Club Rodeo this weekend. Hosted by stock contractor H-Bar-M and sponsored by L&M Motors, the Southern Rough Stock Association-sanctioned rodeo will feature bull riding, barrel racing, mutton bustin', mechanical bulls, stick horse racing, and rodeo clowns. The rodeo will be held Friday, June 2, and Saturday, June 3, in the Layman Building at Athens Regional Park. Gates will open each day at 5:30 p.m., with the show beginning at 8 p.m., rain or shine. "This is always an action-packed event from start to finish," said Mike Moore of H-Bar-M Rodeos. Part of the Ram Rodeo Series, the event will feature "Hollywood Slinger," the bull actor Scott Eastwood rode in the movie "The Longest Ride." A number of events will be held for the little cowgirls and cowboys, including stick horse races and Mutton Bustin'. "Mutton Bustin' is really a big draw for the kids," said Moore. "They love riding those sheep." Two lucky kids will be able to take home their own calf - or cash -during the rodeo. Athens Stockyard will again donate one calf each night during the Calf Scramble, which is open to children … Continue reading

Posted in Spinal Cord Injury Treatment | Comments Off on Chilhowee Shrine Club Rodeo is this weekend – The Daily Post-Athenian

Invivo Therapeutics Holdings Corp (NVIV) Getting Somewhat Favorable Media Coverage, Study Shows – The Cerbat Gem

Posted: Published on May 31st, 2017

Invivo Therapeutics Holdings Corp (NVIV) Getting Somewhat Favorable Media Coverage, Study Shows The Cerbat Gem The Company is engaged in developing and commercializing biopolymer-scaffolding devices for the treatment of spinal cord injuries (SCI). The Company's approach to treating acute SCIs is based on its investigational Neuro Spinal Scaffold implant, ... and more » Go here to read the rest: Invivo Therapeutics Holdings Corp (NVIV) Getting Somewhat Favorable Media Coverage, Study Shows - The Cerbat Gem … Continue reading

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Common acne medication offers new treatment for MS patients say U of C researchers – Calgary Sun

Posted: Published on May 31st, 2017

Calgary Sun Common acne medication offers new treatment for MS patients say U of C researchers Calgary Sun MS is a condition where immune cells get overly activated and can make their way into the brain and spinal cord to produce injury. Similar to the use of minocycline for acne, which prevents inflammation in the skin, it can be used to decrease the ... 'It's a big discovery because it's a cheap, generic, oral medication,' says University of Calgary neurologistCBC.ca all 11 news articles » Go here to see the original: Common acne medication offers new treatment for MS patients say U of C researchers - Calgary Sun … Continue reading

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Soy sauce fungus paves way for cheaper Parkinson’s treatment … – RT

Posted: Published on May 31st, 2017

Published time: 31 May, 2017 17:14 A newly discovered enzyme, found in a fungus used to make soy sauce, could be key to cheaper and more efficient Parkinsons Disease drugs, according to new research. Scientists at the Universities of Manchester and York found the enzyme in Aspergillus oryzae a fungus commonly used to ferment soybeans for sauce. The enzyme, RedAm, could boost monoamine oxidase (MOA) inhibitors used to help Parkinson's sufferers by reducing some of the associated motor symptoms, according to the study published in Nature Chemistry. There is currently no cure for Parkinson's. Around 1 million people in the US live with the degenerative disorder, according to the Parkinsons Disease Foundation. The new biocatalyst will have a positive impact on the costings and manpower needed to produce chiral amines, key to the fight against the disease, according to a statement from the University of Manchester. This is a very exciting discovery from both a chemistry and pharmaceutical perspective, Nick Turner, Professor of Chemical Biology from the Manchester Institute of Biotechnology (MIB) said. It is the first enzyme of its kind that has these properties and has the potential to improve the production of this and other important drugs. Some … Continue reading

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