Category Archives: Muscular Dystrophy Treatment

Liestal, Switzerland, April9, 2015 - Santhera Pharmaceuticals (SIX:SANN) announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Santhera's Raxone/Catena (idebenone) for the treatment of Duchenne Muscular Dystrophy (DMD). FDA's Fast Track program facilitates the development and review of important drugs intended to treat serious conditions and fill an unmet medical need for the purpose of getting them to the patient earlier. Santhera previously announced that the PhaseIII trial (DELOS) in DMD met its primary endpoint and demonstrated that Raxone/Catena delayed the loss of respiratory function. "We are very pleased that the FDA has granted Fast Track designation for Raxone/Catena, which further underscores the unmet medical need for effective treatments for patients with DMD," commented Thomas Meier, PhD, CEO of Santhera. "On the basis of the positive data from our PhaseIII trial with Raxone/Catena in DMD, we have started to prepare a New Drug Application and plan to meet with the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting." About FDA Fast Track Designation The FDA established the Fast Track Drug Development Program under the FDA Modernization Act of 1997. The program is designed to facilitate the development and … Continue reading →

WATERTOWN, N.Y. (Tribune News Service) Some veterans and advocates say New Yorks newly approved medical marijuana rules have left a gap by not allowing medical professionals to provide treatment for post-traumatic stress disorder. The newly finalized state regulations allow medical marijuana in non-smokable forms to treat debilitating or life-threatening conditions such as cancer, HIV/AIDS and Parkinsons disease, but not PTSD, experienced by many in the military. Robert P. Loughhead, veterans outreach coordinator at the Vets Peer to Peer Outreach Center on State Street, said hes heard more feedback from north country veterans in favor of opening access. They dont like the other medications they get from the VA, he said. People dont want to lose all of their emotions. He said his patrons have told him other medications have made them lose their ability to care about things around them. The condition list is just one concern about the state regulations, along with patient access, given the cutoff at 20 dispensaries statewide, and prices that will be set by the state and not the market. Though a state Department of Health assessment indicated an interest by acting Commissioner Dr. Howard A. Zucker in considering other ailments, the current list is … Continue reading →

Treatment on the fast track for 5-year-old INDIANAPOLIS, Ind. (WKRC) -- People facing life-and-death struggles in Indiana have some new hope. Local 12 News reported Tuesday, March 24, about the new Right to Try law. A little boy... By: LOCAL 12 … Continue reading →

Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform to simultaneously modulate multiple targets in a disease pathway, with a focus on diseases like Duchenne muscular dystrophy and ALS The startupsin the midst of a $20.4 million fundraise, according to a recent SEC filing. So far, its raised about $12.4 million in this round. The company says its platform can simultaneously modulate a number of biological targets within several related disease pathways. These are small chemicals designed to join two separate bioactives into a single conjugate molecule, Catabasis says. The platforms called Safely Metabolized And Rationally Targeted, shortened to SMART. The MIT Technology Review wrote up a nice explainer of Catabasis technology two years ago: Catabasis has found a way around the challenge of identifying a single active molecule that can hit multiple pathways effectively: using a chemical linker to bring together two active molecules. The synergistic effect of the linked molecules may arise from the fact that the two compounds both get to the right place at the right time, says [cofounder and CSO Mike] Jirousek. Catabasis has a number of products in its pipeline. Furthest along is its Duchenne muscular dystrophy treatment. … Continue reading →

Muscular Dystrophy | Ayurvedic Treatment | Prof. Dr. Murali Manohar Chirumamilla, M.D. (Ayurveda) For Details and Appointments .... Prof. Dr. Chirumamilla Murali Manohar, M.D. (Ayurveda) Clinic and Consultation Raksha Ayurvedic Centre, H. No. 1320, Ground Floor, (left 6th gate) Opposite... By: Murali Manohar Chirumamilla … Continue reading →

TRIBUTES have been paid to a popular and much-loved teenager who has died after suffering from leukaemia. Matthew Gillespie, 14, a pupil at Tadcaster Grammar School, passed away after being diagnosed with leukaemia which doctors discovered as he was undergoing treatment for muscular dystrophy. On Friday the school paid tribute to Matthew - described as a fun-loving, happy and caring young man - with a non uniform day to raise money for his chosen charities. As Matthew was a big fan of Castleford Tigers and attended many of their matches, it was decided that staff and students at Tadcaster Grammar School would be asked to wear their favourite sports shirt and to donate 1 to do so. Steve Gillespie, Matthew's dad, said: "It's great they have remembered him for the nice person he was. "He was a happy, cheerful lad. He never once complained about his disability. He never asked, 'what have I got? Why me?' He got on with life. "We're happy he is being remembered as the loving, funny young man he was." The Year 10 pupil was diagnosed with muscular dystrophy at the age of five. He was diagnosed with leukaemia in September after undergoing blood tests … Continue reading →

Muscular Dystrophy Treatment in Ayurveda - Mayopathys Home Tirunelveli Muscular Dystrophy Treatment. Magical Therapy - We cure it in a simple Ayurvedic way which otherwise appears as an incurable disease. By: … Continue reading →

Muscular dystrophy only affects young males and is the most common inherited neuro-muscular condition here, affecting one in 3,500 boys Researchers in Dublin and Bonn have made an important discovery related to muscular dystrophy, a relatively common inherited degenerative disorder. The finding could make it possible to diagnose the condition much earlier, and may also point a way towards new treatment options. Muscular dystrophy only affects young males and is the most common inherited neuro-muscular condition here, affecting one in 3,500 boys. Muscle function gradually declines and sufferers have an average life expectancy of just 20 years. Research led by the University of Maynooth with colleagues from the University of Bonn and Dublin City University decided to carry out a detailed study of what was going on inside the muscle cells of those with the condition, said Prof Kay Ohlendieck, chair of Maynooths department of biology. We looked at 1,000 different proteins, comparing those from a healthy cell with those from a diseased cell, looking for any differences between them, he said. The disease interferes with a protein that helps muscle cells to be elastic, which in turn causes a buildup in the cells of a structural protein called collagen. … Continue reading →

CHICAGO, March 26, 2015 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association announced today that thanks to a grant from Genzyme, a Sanofi company, and in collaboration with Emory Genetics Laboratory, MDA clinics are now offering genetic testing for people experiencing muscle weakness suggesting limb-girdle muscular dystrophy (LGMD) who do not already have a genetic diagnosis. This advancement in diagnosis will help more people living with LGMD, and their clinicians, find the most accurate treatment path available. "LGMD is a disorder with more than 30 genetic subtypes. What all the subtypes have in common is weakness of the 'limb-girdle' muscles the muscles surrounding the shoulders and hips," said Valerie A. Cwik, MDA Executive Vice President and Chief Medical and Scientific Officer. "This test will allow clinicians to diagnose and treat individuals with more accuracy as some subtypes require different forms of intervention because they are much more likely than others to involve the cardiac or respiratory muscles." Additionally, weakness of the proximal muscles that appears to be associated with LGMD can occasionally be caused by a metabolic muscle disorder known as Pompe disease, for which enzyme replacement therapy is available. This test will help to accurately diagnose Pompe disease and point patients … Continue reading →

IMAGE:Mani S. Mahadevan, M.D., of the UVA School of Medicine, has identified a potential treatment for the most common form of muscular dystrophy, myotonic muscular dystrophy. view more Credit: Josh Barney | UVA Health System A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could modify progression of muscle damage and muscle dysfunction associated with the disease - issues that cause patients significant disability and deterioration in quality of life. The potential treatment is an experimental drug currently being evaluated for treating other conditions, such as rheumatoid arthritis. In lab tests, mice with myotonic dystrophy that were given the treatment had better muscle function in tests such as running on a treadmill and had improved grip strength. In addition, their muscles became healthier and, notably, many even lived longer. While more testing needs to be done, Mani S. Mahadevan, MD, of the University of Virginia School of Medicine, is hopeful about the drug's potential in humans as well. "The nice thing about this therapy is that we know that it's already been shown to be safe, because clinical trials have already been done with it for other … Continue reading →