Category Archives: Muscular Dystrophy Treatment

What is Muscular Dystrophy ? Muscular dystrophies, or MD, are a group of inherited conditions, which means they are passed down through families. They may occur in childhood or adulthood. There are many different types of muscular dystrophy. They include: Symptoms vary with the different types of muscular dystrophy. All of the muscles may be affected. Or, only specific groups of muscles may be affected, such as those around the pelvis, shoulder, or face. Muscular dystrophy can affect adults, but the more severe forms tend to occur in early childhood. Symptoms include: There are no known cures for the various muscular dystrophies. The goal of treatment is to control symptoms. Physical therapy may help patients maintain muscle strength and function. Orthopedic appliances such as braces and wheelchairs can improve mobility and self-care abilities. In some cases, surgery on the spine or legs may help improve function. The person should be as active as possible. Complete inactivity (such as bedrest) can make the disease worse. This is what you will find so far when you search for a treatment for Muscular Dystrophy on Google or any browser. What i will share with you is not a TREATMENT but what you can … Continue reading →

HACKENSACK, N.J., Dec. 8, 2014 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) announced today that it will award Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and efficacy of an investigational new drug known as HT-100 (delayed-release halofuginone). Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births. HT-100 is an orally available small molecule drug candidate being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne patients. The application of HT-100 to Duchenne and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. Akashi, led by CEO Marc Blaustein, has been granted orphan designation for Duchenne in both the U.S. and EU, as well as Fast Track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals across the U.S. The grant will be used to evaluate the safety, tolerability and pharmacokinetics of HT-100 in patients with Duchenne in both a phase 1b open-label, single and multiple ascending dose study … Continue reading →

NHS chiefs have halted the assessment of a pioneering drug used to tackle a rare muscle-wasting condition. As reported yesterday, NHS England was expected to make a decision this month on whether to fund Translarna, the first treatment developed for sufferers of Duchenne muscular dystrophy. Yet it has now paused assessment of the treatment that was devised with the help of experts at Newcastle University and axed a vital meeting on the drug that was due to take place next week. This is thought to be while it reconsiders how it assesses new drugs. The news has come as a huge disappointment to parents whose child would benefit from the treatment, leading medics and campaign groups backing the groundbreaking drug. Schoolboy George Pegg has been part of the North Easts clinical trial for the past six years. The 11-year-old, of Blyth, was diagnosed with Duchenne muscular dystrophy when he was just three-and-a-half years old and is still able to walk unaided. His parents Lilian, 37, and George, 41, a roofer, have remained as positive as they can for George and their younger son, John, six, who does not have the condition. Mother-of-two Lilian said: It is absolutely devastating for George … Continue reading →

Kate Bushby, leading professor in neuromuscular genetics A leading expert in the North East who has helped pioneer a treatment to tackle a rare muscle wasting condition is urging NHS chiefs to back the groundbreaking drug. This month NHS England is expected to make a decision on whether to fund Translarna, the first treatment developed for sufferers of Duchenne muscular dystrophy. The genetic illness is a progressive disease that is caused by mutations within an individuals DNA that prevents the body from producing the protein dystrophin. This protein forms essential connections in the muscle tissue, and without it muscles degenerate and become weak. Translarna is designed to make the body skip genetic mutations when producing dystrophin. It is only suitable for 10-15% of those with the illness living with a nonsense mutation, which is estimated to be approximately 200 patients in the UK. Earlier this year, the European Medicines Agency gave conditional approval for the drug. It is now up to individual EU member states to decide whether to fund the treatment. Kate Bushby, a leading professor of neuromuscular genetics, has been spearheading a clinical trial of Translarna at The John Walton Muscular Dystrophy Research Centre, which is part of … Continue reading →

KEARNEY, Neb. Sigma Phi Epsilons 6th Annual Muscle Walk event at the University of Nebraska at Kearney is this Thursday December 4 for the Muscular Dystrophy Association (MDA). UNK officials say the Muscle Walk, which will take place in the UNK Cushing Fieldhouse, will include games inspired by activities that children with muscular dystrophy experience while attending MDA summer camps. Sigma Phi Epsilon set a goal of $10,000. Members are raising money by collecting donations, proceeds from donations at the event and business sponsorships. UNK officials say the amount raised by the men of Sigma Phi Epsilon will be matched with a donation from Womack Machine & Supply Company in Texas. The proceeds go to the Omaha headquarters for MDA. Part of the money goes to the national research efforts, and part goes to support local families who have one or more members with muscular dystrophy, said Bryce Clay of Columbus, co- chair of Muscle Walk. UNK officials say Muscular dystrophy is a neuromuscular disease that results in muscle weakness and a decrease in muscle mass. MDA supports cutting-edge research, ensures that Americans affected by the disease receive treatment and critical care, and serves as a voice for families with … Continue reading →

Muscular Dystrophy natural treatment options Feb 19 2014 by Ray Sahelian, M.D. Much has been said about the traditional medical approach to the treatment of muscular dystrophy. On this web page I try to include information on natural supplements that perhaps could be of help to those who have this difficult medical condition. We will update this site as more research is available. Suggestions to discuss with your doctor It may be worthwhile to try small mounts of amino acid supplements and perhaps CoQ10, but these are just suggestions and more research is needed to determine if they are effective and to determine whether additional supplement could be helpful in those with muscular dystrophy. Glutamina or amino acid supplementation for Duchenne muscular dystrophy Glutamine has been shown to acutely decrease whole-body protein degradation in Duchenne muscular dystrophy. To improve nutritional support, we tested whether oral supplementation with glutamine for 10 d decreased whole-body protein degradation significantly more than did an isonitrogenous amino acid control mixture. Twenty-six boys with Duchenne muscular dystrophy were included in this randomized, double-blind parallel study; they received an oral supplement of either glutamine (0.5 g . kg(-1) . d(-1)) or an isonitrogenous, nonspecific amino acid mixture … Continue reading →

By Laith Agha lagha@marinij.com @LaithAgha on Twitter BioMarin agreed this week to buy Dutch biotech company Prosensa Holding N.V., a move intended to expand the Marin-based company's repertoire of drugs that fight rare diseases. Prosensa specializes in drugs for treating muscular dystrophy, a disease that weakens the musculoskeletal system. BioMarin's most immediate interest in the company focuses on the drug drisapersen, which treats Duchenne, a specific form of muscular dystrophy that primarily affects boys. It is under review by the U.S. Food and Drug Administration as is being fast-tracked for approval, BioMarin officials said. "The acquisition of Prosensa fits really well with our mission in delivering treatment in rare diseases," said BioMarin spokeswoman Debra Charlesworth. "This has the potential to be one of our largest commercial products." With the deal still pending, the offer is for $680 million up front, a price based on BioMarin paying $17.75 per share for Prosensa's stock. The deal also includes two additional $80 million payments, one of which will be paid if drisapersen is approved in the United States by May 15, 2016, and the other if the drug is approved in Europe by Feb. 15, 2017. BioMarin, which projects up to $710 million … Continue reading →

BioMarin Pharmaceutical Inc. (BMRN) agreed to pay $680 million to buy Prosensa Holding NV to add a potential treatment for Duchenne muscular dystrophy to its rare-diseases portfolio. BioMarin may also make two payments of about $80 million each if U.S. and European approvals for drisapersen occur by certain dates, the San Rafael, California-based company said today in a statement. BioMarin will pay $17.75 a share for Prosensa, or 55 percent more than its closing share price on Nov. 21. Prosensas drisapersen is a potential first-to-market and best-in-class product for treating a large population of patients with a rare, fatal genetic disease, BioMarin said. The medicine may be appropriate for as many as 10,000 patients, while follow-on products could serve 35,000 people in BioMarins commercial regions, it said. We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible, Chief Executive Officer Jean-Jacques Bienaime said in the statement. The extra milestone payments will come if Prosensas treatment is accepted in the U.S. no later than May 15, 2016, and in Europe no later than Feb. 15, 2017, BioMarin said. The deal will add to the U.S. companys profitability in … Continue reading →

By Estel Grace Masangkay Sarepta Therapeutics announced that it has begun the clinical study of its lead exon-skipping therapeutic candidate eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Eteplirsen is an investigational drug that leverages Sareptas novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology, specifically to skip exon 51 of the dystrophin gene. The drugs mechanism of action aids in the repair of certain gene mutations that affect an estimated 13 percent of the total DMD population. Eteplirsen aims to stabilize or slow the disease process and improve the quality of life of DMD patients. The open-label study 4658-204 (Study 204) will investigate the safety of eteplirsen in patients or those who have late-stage DMD and fail a minimum 6-minute walk test score at baseline. The company said it will enroll around 20 patients treated with eteplirsen who have genotypes likely to be responsive to exon 51 skipping and who otherwise qualify for the study. Edward Kaye, CMO of Sarepta, said, The initiation of this eteplirsen study represents an important milestone for patients, their families, and the DMD community. Expanding the DMD population to include patients who are older and non-ambulant will provide additional data to support … Continue reading →

Clinical Study Intended to Identify Novel microRNA Candidates for the Treatment of Duchenne Muscular Dystrophy PRINCETON, NJ and REHOVOT, ISRAEL and BOTHELL, WA--(Marketwired - Nov 18, 2014) - Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based molecular diagnostics and therapeutics, and Marina Biotech, Inc. (OTCQB: MRNA), a leading nucleic acid-based drug discovery and development company focused on rare diseases, jointly announced the initiation of the first project under the Strategic Alliance the companies announced on April 2, 2014.The first project will be a clinical study conducted by Rosetta Genomics to identify novel microRNA candidates for the treatment of Duchenne Muscular Dystrophy (DMD). In this study, Rosetta will evaluate archived muscle biopsy samples from 10-30 DMD patients and 10-30 non-DMD controls. The samples will be processed and assessed by Rosetta's microarray platform. Samples will also be profiled using deep sequencing to identify novel microRNAs and isomiRs. "We are excited to initiate our strategic agreement with Marina Biotech on this important project in DMD, as it is a debilitating disease of significant unmet need," stated Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics."We look forward to leveraging our microRNA biomarker platform for the discovery of … Continue reading →