Category Archives: Muscular Dystrophy Treatment

Duchenne Trial Top-Line Results In April, Capricor reported more positive six-month top-line results from the Phase I/II Halt cardiomyOPathy progrEssion in Duchenne (HOPE-Duchenne) clinical trial, designed to assess the safety and preliminary efficacy of CAP-1002 in 25 subjects. Capricor cited statistically significant improvements in systolic thickening of the inferior wall of the heart and in the function of the middle and distal upper limb in CAP-1002 patients compared with control patients receiving usual care. The HOPE-Duchenne trial also found differences in several other cardiac and skeletal muscle measures, including cardiac scar, that Capricor said were consistent with a treatment effect. "Over the last few years, and during the term of the Janssen option period, we believe that significant value for our CAP-1002 asset has been created through the demonstration of clinical proof-of-concept to treat DMD, and also from the progress that has been made toward the development of a commercial-scale manufacturing process for the cells," Capricor president and CEO Linda Marbn, Ph.D., said in the statement. As a result, she said, Capricor will continue to develop CAP-1002, either alone or with a new collaboration partner. After announcing HOPE results, Capricor discussed potential product registration strategies for this indication at a … Continue reading →

The Republican health-care bill thats already encountering stiff opposition from hospitals, doctors, and patients now has another group worried: rare-disease drugmakers and the patients who need those high-priced treatments. The proposal, which the Senate could vote on as soon as next week, would give states leeway to allow the return of health coverage caps -- called lifetime limits -- that were banned under Obamacare. That could allow insurers, including those that offer plans through employers, to cut off reimbursements once an insured person spends $1 million to $2 million on prescription drugs. With expensive rare-disease drugs, that can happen quickly. Treatments for rare diseases brought in $114 billion in worldwide sales last year,according to Evaluate Ltd., a life sciences data company, and drugmakers big and small are increasingly banking on them to boost revenue. If insurers are permitted to stop paying for them at a certain point, the burden may fall to patients, and ultimately drug companies will pick up the tab for those who cant afford the cost, said Steve Holtzman, chief executive officer of closely-held Decibel Therapeutics Inc. Where a patient exhausts the limit and cant pay, youre using the patient assistance programs and providing the drug for … Continue reading →

June 28, 2017 A cross section of a muscle biopsy: Blood vessels (red) between the muscle fibers and laminin-2 (green), which surrounds each muscle fiber. Credit: University of Basel, Biozentrum The cell scaffolding holds muscle fibers together and protects them from damage. Individuals who suffer from muscular dystrophy often lack essential components in this cell scaffold. As a result, their muscles lack strength and become progressively weaker. The research team of Prof. Markus Regg at the Biozentrum, University of Basel, has now designed two proteins that stabilize the cell scaffolding link it to the muscle fiber and thereby restore muscle structure and function. Their findings are published in the current issue of Science Translational Medicine. Muscular dystrophy is a term used to describe many different muscular diseases caused by genetic defects. To date, there are no treatments available to stop disease progression. In their study, the scientists led by Prof. Markus Regg have investigated a specific type of muscular dystrophy, called congenital muscular dystrophy. In an animal model, they demonstrated for the first time that two proteins designed by the researchers not only recover muscle force and increase body weight in the sick animals but also significantly prolong survival. Severe … Continue reading →

June 22, 2017 01:23 ET | Source: Santhera Pharmaceuticals Holding Ltd multilang-release Liestal, Switzerland, June22, 2017 - Santhera Pharmaceuticals (SIX:SANN) announces that the UK's Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for patients with respiratory function decline not taking glucocorticoids in Duchenne Muscular Dystrophy (DMD). The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.1 The MHRA decision allows patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for DMD for Marketing Authorization by the European Medicines Agency (EMA). Under the EAMS, and as shown in the public assessment report,2 Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in … Continue reading →

A drug formerly meant for cancer treatment may be better used to treat muscular dystrophy, according to researchers from the National Institutes of Health (NIH) and the University of Nevada, Reno School of Medicine. Known as SU9516, the drug was meant to be used to battle leukemia until researchers discovered that it ramped up production of a protein missing from patients with muscular dystrophy, a disease that causes muscles to deteriorate. Although the drugs positive results have only been confirmed in animal testing, scientists are hoping the discovery will provide the missing link for better muscular dystrophy treatment. The studys chief author, pharmacology professor Dean Burkin of the University of Nevada, said he thinks SU9516 and other medicines could also treat illnesses other than muscular dystrophy. Integrin stabilizes muscle structure, and helps stimulate muscle repair and regeneration, Burkin said in the release. If we can artificially increase its production with drugs, we think it can help protect muscle cells from damage. Read more here: Potential leukemia drug better aimed at muscular dystrophy, NIH says - Patient Daily … Continue reading →

David Cox pictured with his three kids when he was working for the Lynchburg Fire Department. ALTAVISTA, Va. (WSET) -- David Cox has been fighting fires and helping save lives for 15 years with the Lynchburg Fire Department. Cox says it's been a life-long dream and has always been his passion. But his wife Bess Cox said a of couple months ago, he received the worst news of his life. "As of right now he has been diagnosed with Myasthenia Gravis, which is a form of Muscular Dystrophy," She said. So now instead of fighting fires, he is fighting for his own life. Calvary Baptist Church wanted to step in and help the Cox family once word got around their town of Altavista about what he was going through. Cox and his wife has three kids. Pastor Seth Peterson decided to put together a basketball tournament and barbecue to raise money for Cox's medical bills. He said their goal was to raise $5,000, but they surpassed that goal and raised around $6,500. Calvary Baptist Church is not even the Cox's home church, but have a few friends that are members. Pastor Peterson said their church just wanted to help a … Continue reading →

About Duchenne Muscular Dystrophy Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms. DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. It is estimated that a nonsense mutation is the cause of DMD in approximately 13 percent of patients. About ataluren (Translarna)Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is … Continue reading →

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with Frances Genethon, a nonprofit R&D org. The research collaborationwill see the Franco-American pair jointly develop treatments for Duchenne muscular dystrophy and comes after Sareptas first FDA approval for DMD with its controversial med Exondys 51 (eteplirsen). RELATED: FDA expert lashes out at 'worrisome' Sarepta approval in JAMA Sarepta is looking to tap into Genethons preclinical microdystrophin gene therapy approach, which can target the majority of patients with DMD. Its current med can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD. It is hoping that with new tie-ups, it could produce a gene therapy that could treat many more, if not all, patients with the disease, although this is still some years off. DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boysand occurs in about one out of every 3,600 male infants worldwide. This builds on the pacts announced at the start of the year at the JPM conference, which saw it sign a … Continue reading →

Muscular Dystrophy News Researchers Aim to Repurpose Failed Leukemia Drug to Treat ... Managed Care magazine Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and at the University of Nevada Reno School ... A Drug Unsuccessful at Treating Cancer May Successfully Treat DMDAJMC.com Managed Markets Network Duchenne MD Mice Improve Muscle Function with Former Cancer ...Muscular Dystrophy News all 3 news articles » See the original post here: Researchers Aim to Repurpose Failed Leukemia Drug to Treat ... - Managed Care magazine … Continue reading →

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne. In these models, the RASRx1902 has improved muscle strength and regeneration while decreasing muscle inflammation, degeneration, and necrosis. Through this ODD, RASRx is eligible for financial incentives that can stimulate investment in this program and expedite its regulatory timeline. In concert with CureDuchenne, the company is now transitioning to toxicology studies to advance RASRx1902 towards human clinical trials for the treatment of Duchenne. RASRxs founders worked with the USC Stevens Center for Innovation, the technology transfer office for the University of Southern California, to exclusively license RASRx1902. Funding by CureDuchenne Ventures and a collaborative U.S. Department of Defense research grant with USC has accelerated the preclinical development of this program. Innovation is a core part of USCs culture and we are proud to support Dr. Kathleen Rodgers and RASRx to facilitate the technology transfer of RASRx1902 for DMD, said Michael Arciero, J.D. Director of Technology Commercialization and New Venture, … Continue reading →