Category Archives: Muscular Dystrophy Treatment

Duchenne muscular dystrophy (DMD) is one of the most common types of muscular dystrophy, which is an X-linked inherited genetic disorder characterized by gradual skeletal muscle weakness. According to NHS, in every year 100 boys are born with Duchenne muscular dystrophy in the U.K. As per DMD surveillance report by Center for Disease Control and Prevention (CDC), 349 new cases of Duchenne muscular dystrophy were reported in 2010 in the U.S. Duchenne muscular dystrophy usually affects male child and first signs and symptoms of the disease are observed at the age of 2-3 years. Duchenne muscular dystrophy worsens quickly which results in loss of ability to walk and eventually causes death within 20-30 years of age. Currently there is no curative treatment for Duchenne muscular dystrophy however symptomatic treatment approaches such as pain alleviating drugs and steroids to slow down the progression of disease are recommended by majority of physicians. Treatment is mainly aimed to maximize the quality of life. A sample of this report is available upon request @ http://www.persistencemarketresearch.com/samples/12475 Continuous research on gene therapy, exon skipping drugs and stem cell therapy for the treatment of Duchenne muscular dystrophy is expected to fuel the global market for Duchenne muscular … Continue reading →

SYDNEY, April 4, 2017 /PRNewswire/ --Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is pleased to announce that the initial pre-clinical efficacy results of the OPMD program have been published in Nature Communications, an open access scientific journal published by the Nature Publishing Group. OPMD, a rare progressive muscle-wasting disease caused by mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, is characterised by eyelid drooping, swallowing difficulties, and proximal limb weakness. A direct link to the article can be found on Nature's website http://www.nature.com/articles/ncomms14848.epdf The key results from these studies demonstrate that a DNA directed RNA interference (ddRNAi) approach to 'silence and replace' the mutant PABPN1 protein, results in the correction of the muscular dystrophy and of key clinical features of OPMD including a progressive atrophy and muscle weakness associated with nuclear aggregates of insoluble PABPN1. These data were generated in the A17 mouse model that expresses the mutant PABPN1 gene and mimics most of the features of human OPMD patients. These findings were central in being able to receive the Orphan Drug Designation in the European Union for the OPMD program in January of this year. "These published results have been critical for establishing the proof of … Continue reading →

Bangladeshi father's cry for euthanasia prompts Indian clinic to treat patients pro bono Times Now Dhaka: Three Bangladeshis suffering from a highly debilitating form of muscular dystrophy are receiving free treatment in India after a relative's plea for them to be allowed to die brought their plight to global attention. Fruit seller Tofazzal ... and more » Read the rest here: Bangladeshi father's cry for euthanasia prompts Indian clinic to treat patients pro bono - Times Now … Continue reading →

Mumbai, Apr 4 (PTI) Three Bangladeshi patients, suffering from an incurable muscular dystrophy, today underwent first stem cell therapy at a Navi Mumbai based treatment centre. "We applied a simple therapy to the patients. We took out the stem cells from their bone marrow in the hip bone and after the required processing we injected it back into their body. "We will wait for the results as to how they respond to such treatment. Meanwhile, physiotherapy and occupational therapy is being offered to them as well," Avantika Patil, coordinator between the patients and the treating centre told PTI. Patil, is part of the team of NeuroGen, a brain and spine institute that keeps the track of its patients. "We learnt about the patients through an article in an international newspaper and decided to contact them as we specialise in treating such diseases. "I am also in contact with one Noor Khan from Bangladesh, an activist who helped the three patients to furnish documents and visa procedures," she said. A Mumbai-based organisation specialising in such diseases, Meditourz, in collaboration with NeuroGen based in Navi Mumbai, offered to provide treatment to them. The trio have been suffering from a rare disease Duchenne … Continue reading →

What happened Shares of Australian healthcare company Benitec Biopharma (NASDAQ:BNTC) rose almost 80% this morning after the company announced that it had published data in Nature Communications, an open-access scientific journal, supporting its novel approach to treating oculopharyngeal muscular dystrophy, or OPMD. As of 11:45 a.m. EDT, the microcap stock had settled down to a 55% gain. The preclinical study demonstrated that faulty genes at the root of OPMD could be silenced and then replaced with working copies through a gene-editing technique known as DNA directed RNA interference, or ddRNAi. Mice that underwent the gene replacement therapy displayed several important benefits including a reduction in muscle fibrosis (the scarring of connective tissue) and returning to normal muscle strength. Image source: Getty Images. The data from the preclinical mouse model study clearly have investors looking ahead to the prospects of clinical trials with human patients. That's not exactly a stretch, either. In January the company gained Orphan Drug Designation from the European Union using the very same preclinical results and will look to move into a phase 1 trial in the near future. Orphan Drug Designation is applied to drugs treating rare diseases and grants special additional protections if the treatment … Continue reading →

April 03, 2017 08:30 ET | Source: Sarepta Therapeutics CAMBRIDGE, Mass., April 03, 2017 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, today announced the appointment of Catherine Stehman-Breen, M.D., M.S., as chief medical officer. Dr. Stehman-Breen joins Sarepta after having served as vice president, clinical development and regulatory affairs at Regeneron Pharmaceuticals since 2015, initially as head, pain therapeutic area, and subsequently as head, clinical project management and operations. From 2003 to 2015, she held senior leadership roles at Amgen including vice president, global development, leading the neuroscience, nephrology and bone therapeutic areas. I deeply admire Sareptas profound commitment to improving the lives of boys with Duchenne muscular dystrophy and the exciting and innovative PMO and PPMO platform that is being harnessed to achieve this goal, said Dr. Stehman-Breen. I am excited to join the company at a time when it is rapidly building and look forward to working closely with the internal team and external collaborators as we seek to develop and commercialize novel therapies that address this significant unmet medical need. We are thrilled to have Dr. … Continue reading →

Showing its humane face, national carrier Air India on Sunday flew three young Bangladeshi citizens, suffering from serious muscular dystrophy, for treatment at a Mumbai hospital along with their relatives -- free of charge. The three patients and three persons accompanying them took Air India flight from Kolkata to Mumbai on Sunday evening, and will also return by an Air India flight after treatment -- without having to pay any fare. AI-773 took off from Kolkatas Netaji Subhas Chandra Bose International Airport with the six Bangladesh nationals among other passengers -- bringing a ray of hope to the patients suffering from a rare medical condition called Duchenne Muscular Dystrophy, an Air India media release said. The patients were extended full support by way of wheelchairs and individual attendants. They, along with their relatives, were handed over the boarding cards by senior Air India officials. The saga of Abdus, Rahinul and Shorab aged 24, 14 and 8, respectively, goes back to their birth when they were diagnosed with this crippling disease. Read more It grabbed attention when their family pleaded with the Bangladesh government for mercy killing of the three for failing to continue with their treatment due to financial reasons. … Continue reading →

13News Now Niko Clemmons has the story Niko Clemmons, WVEC 10:22 PM. EDT March 29, 2017 YORKTOWN, Va. (WVEC) -- A Yorktown mother and her son are fighting for what they believe is right. We first told you about Caden Bowers back in September when he got a chance to score a touchdown for Tabb Middle School. It was the highlight of his life. Caden was born with Duchenne Muscular Dystrophy, but he wasnt diagnosed until he was 5 years old. Duchenne Muscular Dystrophy, or DMD is one of nine types of muscular dystrophy. The disease primarily affects boys, but in rare cases it can affect girls. Symptoms can start showing in people as young as three years old. In the early stages, DMD affects the shoulder and upper arm muscles and the muscles of the hips and thighs. That makes is hard to get up from the floor, climb stairs, or raise your arms. Now Caden's mother says he is now in the middle of another difficult situation. She says their insurance company is refusing to cover a breakthrough treatment. A few months after being diagnosed Caden was one of 12 boys selected for a first-of-its-kind treatment for the … Continue reading →

MINNEAPOLIS (KMSP) - University of Minnesota research focused on a specific type of muscular dystrophy is giving patients renewed hope. Basically each year you find you lose more and more ability to lift, to walk," said Marge Brchan. Diagnosed at 19 years old,Brchan has battled the effects of facioscapulohumeral muscular dystrophy or FSH most of her life. It was very hopeless, said Brchan. "They knew very little about the disease. There wasn't anyone focusing on the disease. The genetic muscle weakening condition is among the common forms of muscular dystrophy, yet according to the FSH Society is largely unknown to the public or even to most doctors. Patients with this disease, one of the saddest things that happens they are robbed of their ability to smile, said Professor Michael Kyba. Kybaand his staff at the University of Minnesota are doing grant driven research now that the gene causing the disease has been identified. The hope comes down to there is actual work being done to discover the drug. About 10 years ago, we didn't know what gene caused this disease, said Kyba. That's our hope that someday in the near future we'll have candidates that we can actual take to … Continue reading →

Research to find treatments, alleviate patients stress by Nisha Dabhi | Mar 23 2017 | 23 hours ago Profs. Silvia Blemker and Shayn Peirce-Cottler in the Department of Biomedical Engineering received a $2.5 million grant from the National Institutes of Health. The grant will fund research on therapeutic outcomes for individuals with Duchenne Muscular Dystrophy through multi-scale computer modeling techniques. DMD is a devastating but rare musculoskeletal genetic disorder, prevailing at a rate of one in every 3,500 male births. Individuals with DMD appear healthy for the first few years of their lives, Blemker said. However, around the age of three or four, symptoms begin to appear. Patients with DMD typically die around their early thirties. First, the muscles of the hip, pelvic area, thighs and shoulders become fatigued, and then skeletal muscles of the arms and legs begin to deteriorate. In even later stages, the disease affects heart and respiratory muscles impeding normal cardiovascular and breathing functions. DMD is caused by a mutation in the gene encoding dystrophin. Dystrophin is a large protein that supports muscle fiber strength and connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix and links actin filaments to other support proteins. … Continue reading →