Category Archives: Muscular Dystrophy Treatment

By Dow Jones Business News, March 17, 2014, 07:28:00 PM EDT By John Kell Shares of Prosensa Holding NV ( RNA ) jumped Monday after the Dutch biotechnology company reported "encouraging" data from a Phase II study for a Duchenne muscular dystrophy treatment. The compound, drisapersen, is in development to treat DMD, a rare, fatal muscular disorder. DMD is one of the most prevalent rare genetic diseases globally, according to Prosensa, which said the disorder affects as many as 1 in 3,500 boys. The disorder is almost always fatal, and there is no approved disease-modifying therapy. On Monday, Prosensa disclosed data from a 48-week U.S. study in 51 boys with DMD. The results indicated that boys in the higher-dose drisapersen group experienced stabilization and even improvements in their muscle function and physical activity for the 24-week treatment period and maintained that improvement during the 24-week follow-up phase. "The maintenance of the clinically meaningful treatment benefit in the 24-week follow-up phase is very encouraging evidence for the drug's ability to produce prolonged stabilization of disease," said Craig M. McDonald, principal investigator and a professor of pediatrics at the University of California. Investors cheered the news, sending Prosensa's shares up 34% to … Continue reading →

--------------------------------------------------------------------- Prosensa Committed to Defining a Path Forward for Its Lead Product, Drisapersen LEIDEN, The Netherlands, 2014-03-18 12:00 CET (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (Nasdaq:RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported financial results for the full year ending December 31, 2013, and provided an update on the next steps for its exon-skipping platform for the treatment of Duchenne Muscular Dystrophy (DMD). 'As we have previously stated, we are encouraged by initial findings from further analyses of the aggregate drisapersen data that suggest that treating earlier in DMD and treating longer shows a delay in the progression of the disease. These data give us the confidence to engage patient groups, clinical experts and regulators to explore a path forward for drisapersen,' said Hans Schikan, CEO of Prosensa. Mr. Schikan continued, 'While the past few months have been a very important transition period for Prosensa, our primary focus has not changed, which is to improve the lives and outcomes of boys with DMD. At the start of the year we regained the rights to drisapersen and retained rights to our other development-stage compounds for DMD from GlaxoSmithKline (GSK). We are … Continue reading →

Acceleron Pharma Inc.'s (XLRN: Quote) collaboration partner, Celgene Corp. (CELG: Quote) is scheduled to present interim data from an ongoing phase 2a study of Sotatercept in patients with end stage renal disease on hemodialysis at the National Kidney Foundation 2014 Spring Clinical Meeting. The meeting will take place on April 22-26, 2014. XLRN closed Monday's trading 6.08% lower at $42.50. Alphatec Holdings Inc. (ATEC: Quote) said that its consolidated net revenues for full year 2013 rose to $204.7 million from $196.3 million in 2012. Looking ahead to 2014, the company anticipates annual revenues of $208 million to $215 million, representing approximately 1.6% to 5% growth over 2013. The company also noted that it has settled the OrthoTec vs. Surgiview litigation. Under the terms of the settlement, Alphatec has agreed to pay OrthoTec $49 million over the next 7 years in cash, with an initial cash payment of $17.5 million. ATEC closed Monday's trading 15.11% higher at $1.60. In after-hours, the stock gained another 4.37% to $1.67. Amgen Inc. (AMGN: Quote) announced that a phase III trial evaluating its investigational drug Evolocumab has met its primary endpoint of low-density lipoprotein cholesterol reduction in patients with homozygous familial hypercholesterolemia. In the trial, … Continue reading →

Contact Information Available for logged-in reporters only Newswise If you are affected by Malignant Hyperthermia, Congenital Muscular Dystrophies or Congenital Myopathy Subtypes, the Malignant Hyperthermia Association of the United States and the Cure Congenital Muscle Disease organization are asking for your help to enable research and clinical trials by registering with the Congenital Muscle Disease International Registry (CMDIR). You do not have to be diagnosed with a specific muscle disorder to register. We welcome everyone with congenital or later onset muscle weakness to register, and can provide information on institutions who perform genetic testing, the names of medical providers who specialize in the diagnosis and treatment of muscle disease, and community support groups to put you in touch with others affected by a muscle disorder. When you register, you will receive the CMDIR newsletters, notice of available clinical trials that apply to your registered profile, and notice of available therapies that apply to your registered profile when they become available. To register, click this link today and help the CMDIR continue working towards future treatments and a cure! What is Malignant Hyperthermia Malignant Hyperthermia (MH) is inherited genetic disorder found in an estimated 1 out of 2,000 people and triggered … Continue reading →

Maxx Kirley, 12, gets so exhausted walking across his school that he cant participate in gym class once he gets there. He has Duchenne muscular dystrophy, a disease that weakens his muscles. But University research on certain poloxamers, named molecular Band-Aids, could provide a treatment option for Kirley. The groups of compounds can mend muscle cells with no side effects. University researchers are trying to apply these molecular Band-Aids to muscular dystrophy and heart disease, and are planning to start a clinical trial once they decide on the best version of the molecule to use. Having Duchenne muscular dystrophy means that Kirley lacks a large protein called dystrophin in his muscle cells. In healthy individuals, dystrophin absorbs the impact of everyday activities so that muscle cells can repair damage quickly, said Dr. Peter Karachunski, a pediatric neurologist at Amplatz Childrens Hospital. Without the protein, muscles deteriorate over time and even walking is strenuous, he said. The disease can lead to damage in almost every body system, and the life expectancy of a patient is about 20 years. He, first and foremost, is just a 12-year-old boy, Maxxs mother Terri Kirley said. Hes very smart, hes a student and he wants … Continue reading →

Dysphagia facts Dysphagia means difficulty swallowing. Swallowing is a complex action involving the muscles and nerves within the pharynx and esophagus, a swallowing center in the brain, and nerves that connect the pharynx and esophagus to the swallowing center. Dysphagia should be differentiated from odynophagia and globus sensation. General causes of dysphagia can be grouped as either oropharyngeal or esophageal. Specific causes include physical obstruction of the pharynx or esophagus, diseases of the brain, diseases of the smooth muscle of the esophagus, diseases of skeletal muscle of the pharynx, and miscellaneous diseases. Symptoms of dysphagia may be swallowing-related or non-swallowing-related. The differential diagnosis of dysphagia includes odynophagia and globus sensation, tracheo-esophageal fistula, rumination syndrome, gastroesophageal reflux disease (GERD), and heart disease. Evaluation and diagnosis of the cause of dysphagia includes history, physical examination, endoscopy, X-rays, esophageal manometry, esophageal impedence, esophageal acid testing, and tests for the diagnosis of muscular dystrophy and metabolic myopathies. The treatment of dysphagia depends primarily on its cause. Newer diagnostic procedures for the evaluation and diagnosis of the cause of dysphagia include high resolution or 3D manometry and endoscopic ultrasonography. What is dysphagia? Dysphagia is the medical term for the symptom of difficulty swallowing, derived from … Continue reading →

More than 300 runners ready to help fight muscle-wasting conditions 5:24pm Wednesday 5th March 2014 in News By Barry Nelson, Health Editor Runners pictured at the start of the Muscular Dystrophy Campaign Oxford Town and Gown 10K last year. The event is the model for this Sunday's run in Durham City. MORE than 300 runners are ready to take on the first Muscular Dystrophy Campaign Durham City Town and Gown 10k this Sunday and help fight muscle-wasting conditions. The new race follows a route past iconic Durham sights, including the cathedral and castle, leading runners along the banks of the River Wear into the heart of the city. Thanks to fantastic support from locals and Durham University students alike, the event is on course to raise 10,000 towards the Muscular Dystrophy Campaigns work. The charity funds groundbreaking research into treatments for muscular dystrophy and related neuromuscular conditions and supports the 70,000 families affected 3,000 of them from the North-East. The run will be joined by a team of 12 runners from the elite research team at the Institute of Human Genetics at Newcastle University, which is supported by the Muscular Dystrophy Campaign. The innovative work carried out by 70 experts … Continue reading →

HASTINGS Mothers love their children, but when their children have a life-threatening medical condition, their hearts expand so they can perform deeds they never dreamed about. That is the case for Betty Vertin of Hastings, who has become an advocate for awareness, fundraising and research into Duchenne muscular dystrophy, a disease that only allows its victims the briefest taste of life before stealing it away. An online service is needed to view this article in its entirety. You need an online service to view this article in its entirety. Or, use your linked account: Need an account? Create one now. Or, use your linked account: kAm'6CE:? 2?5 96C 9FD32?5[ y2D@?[ 92G6 7:G6 49:=5C6?[ H9:49 :?4=F56D @?6 52F89E6C 2?5 7@FC D@?Di `aJ62C@=5 {6I:[ gJ62C@=5 |2I[ eJ62C@=5 r92?46[ dJ62C@=5 #@H6?[ 2?5 bJ62C@=5 r92C=:6[ H:E9 |2I[ #@H6? 2?5 r92C=:6 2== 5:28?@D65 H:E9 sF496??6 >FD4F=2C 5JDEC@A9J]k^Am kAm'6CE:? D2:5 E92E |2I H2D 5:28?@D65 H:E9 sF496??6 |s H96? 96 H2D c[ H96? x H2D AC68?2?E H:E9 @FC =2DE 49:=5] $96 D2:5 E96 5@4E@C H9@ E2= >F49 9@A6[ E6==:?8 E96> E92E sF496??6 |s 😀 2 72E2= 5:D62D6 H:E9 ?@ 4FC6]k^Am kAm'6CE:? D2:5 D96 :?:E:2==J 6?E6C65 2? :?E6?D6 8C:6G:?8 A6C:@5[ >@FC?:?8 2== E96 E9:?8D D96 2?5 y2D@? H@F=5 … Continue reading →

Ilan Ganot, a hedge fund banker at JPMorgan Chase & Co. (JPM), left his life in finance to create a new business model aimed at developing drugs more quickly. His sons life may depend on his success. Since his 2-year-old was diagnosed a year ago with Duchenne muscular dystrophy, a fatal genetic disease, Ganot quit his job in London, moved his family to the Boston area, raised $17 million and founded a biotechnology company. Ganot, 40, a former Israeli army captain, is one of a generation of parents who are stepping in when medicine and industry dont move fast enough to save their children. Instead of the usual approach of forming or supporting an advocacy group, Ganot is using his roots in finance to start a business aimed at getting treatments to market. Hes a really exceptional example of a parent who says, Im going to take my skills and apply them to this problem, said Sharon Hesterlee, vice president of research at Parent Project Muscular Dystrophy, a nonprofit focused on the disease. You want them to solve world peace after this. Ganot would settle for making a difference in the disease afflicting his son, Eytani. Theres no cure for … Continue reading →

Article Highlights: by Margaret Wahl on February 14, 2014 - 11:59am Children and adolescents with Ullrich congenital muscular dystrophy or Bethlem myopathy are invited to participate in a study of a daily breathing treatment regimen using a brand-name device called a CoughAssist. MDA classifies Ullrich congenital muscular dystrophy as a type of congenital muscular dystrophy (CMD) and Bethlem myopathy as a type of limb-girdle muscular dystrophy (LGMD). Both are caused by genetic mutations that affect collagen 6, a protein that's normally present in the tissue surrounding muscle fibers. Mutations in the same genes have been known to cause either CMD, with muscle weakness beginning at or near birth, or LGMD, with muscle weakness beginning later in life. About the CoughAssist study This is a randomized research study in which participants will be assigned into one of two groups by a computer, not by choice. Half the study participants will use a CoughAssist device with settings determined by specific testing at the research visits. Instructions will be to do breathing exercises twice a day. The other half of study participants will continue their usual respiratory treatments. The CoughAssist machine, made by Philips Respironics, is designed to facilitate clearing respiratory tract secretions. … Continue reading →