Category Archives: Muscular Dystrophy Treatment

WEDNESDAY, Oct. 17 (HealthDay News) -- A man with Duchenne muscular dystrophy who received a device to help his heart's left ventricle pump blood throughout his body could represent a breakthrough in the treatment of the disease, according to his doctors. Jason Williams, 29, of Peebles, Ohio, is believed to be one of the first U.S. patients with Duchenne muscular dystrophy to receive an implanted ventricular assist device, the Cincinnati Children's Hospital Medical Center team said. Ventricular assist devices are mechanical pumps implanted in the chest to help a weakened heart pump blood to the rest of the body. Eighty percent of boys and men with Duchenne muscular dystrophy die of heart failure, according to a medical center news release. Due to the severity of their muscle disease, they are not typically candidates for heart transplant or some other treatment options available to patients with other types of muscular dystrophy. "This is a major milestone in the care of Duchenne muscular dystrophy," Dr. John Lynn Jefferies, director of the heart failure and ventricular assist device programs at the Cincinnati Children's Heart Institute, said in the news release. "This treatment offers the possibility to change the outcome and the lives of … Continue reading →

Public release date: 23-Oct-2012 [ | E-mail | Share ] Contact: Bryan Ghosh bghosh@plos.org 44-122-344-2837 Public Library of Science Boosting the activity of a vitamin-sensitive cell adhesion pathway has the potential to counteract the muscle degeneration and reduced mobility caused by muscular dystrophies, according to a research team led by scientists at the University of Maine. The discovery, published 23 October in the open access journal PLOS Biology, is particularly important for congenital muscular dystrophies, which are progressive, debilitating and often lethal diseases that currently remain without cure. The researchers found that they could improve muscle structure and function in a zebrafish version of muscular dystrophy by supplying a common cellular chemical (or its precursor, vitamin B3) to activate a cell adhesion pathway. Muscle cells are in themselves relatively delicate, but derive important additional mechanical strength from adhesion protein complexes; these anchor the muscle cells to an external framework known as the basement membrane, thereby helping to buffer the cells against the extreme forces that they experience during muscle contractions. Mutations in the genes that encode these adhesion proteins can weaken these attachments, making muscle cells more susceptible to damage and death. The resulting muscle degeneration can eventually lead to … Continue reading →

Although he made it to 29 years old with Duchenne muscular dystrophy, Jason Williams was not expected to survive more than another year. But this week, doctors announced that Williams no longer has an expiration date. Thanks to a groundbreaking surgery that's never been performed on a Duchenne patient in the United States before, there's no telling how many more birthdays -- and Cincinnati Reds seasons -- he'll be around for. "The most important thing this device has given him is hope, because without it, we know what's going to happen," said heart surgeon Dr. David Morales, who implanted Jason's Left Ventricular Assist Device, or LVAD, on Sept. 5 with a team of doctors at Cincinnati Children's Hospital. Duchenne muscular dystrophy is an inherited disease characterized by progressive muscle weakness because of a mutated protein gene. It only affects boys, and usually sets in when the child is between 2 and 6 years old, first confining the child to a wheelchair and ultimately weakening his heart and killing him by his late teens or early 20s. "He's going to be with us longer than we ever anticipated he would be," said Williams's mother, Karen Jones, as she stood next to … Continue reading →

CINCINNATI, Oct. 16, 2012 /PRNewswire-USNewswire/ -- In what may be the biggest breakthrough in muscular dystrophy in years, a patient at Cincinnati Children's Hospital Medical Center is believed to be the first in the nation with Duchenne muscular dystrophy to have a device implanted to help his heart pump blood to the body long-term. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Because 80 percent of boys and men with Duchenne muscular dystrophy (DMD), ultimately die of heart failure, the implanted left ventricular assist device (LVAD), could add years or even decades to the lives of patients with DMD. "This is a major milestone in the care of Duchenne muscular dystrophy," says John Lynn Jefferies, MD, director of the heart failure and ventricular assist device programs at the Cincinnati Children's Heart Institute. "This treatment offers the possibility to change the outcome and the lives of these young men in a significant way that has never been realized up until now." Due to their severe muscle disease, patients with DMD are not typically candidates for heart transplant and some other treatment options that exist for patients with other muscular dystrophies. Patients with Becker muscular dystrophy, for example, a less severe form of muscular dystrophy caused by the … Continue reading →

(RTTNews.com) - Sarepta Therapeutics Inc. (SRPT) said Friday that data from a Phase IIb study of its investigational drug eteplirsen for the treatment of boys with Duchenne muscular dystrophy will be presented on October 13 at the World Muscle Society in Perth, Australia. The presentation will describe new and previously reported efficacy and safety data from the Phase IIb study examining 48 weeks of treatment with eteplirsen in boys with Duchenne muscular dystrophy, or DMD. Results from the Phase IIb extension study confirmed that eteplirsen met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test over the placebo/delayed treatment cohort. Additional data to be presented includes: individual patient data on the primary endpoint of change in dystrophin-positive fibers from baseline; additional biochemical findings including RT-PCR and western blot images from selected patients; additional information on the two patients in the 30 mg/kg cohort who showed a rapidly progressive decline on the 6-minute walk test and were excluded from the analysis. Data to be presented will also include summary of treatment-emergent adverse events comparing eteplirsen-treated patients versus placebo, which demonstrated that eteplirsen was well-tolerated through 48 weeks … Continue reading →

CAMBRIDGE, MA--(Marketwire - Oct 12, 2012) - Sarepta Therapeutics ( NASDAQ : SRPT ), a developer of innovative RNA-based therapeutics, announced today that data from a Phase IIb study evaluating eteplirsen, an investigational treatment for boys with Duchenne muscular dystrophy (DMD), will be presented Saturday, October 13th at the World Muscle Society in Perth, Australia.Principal investigator, Jerry R. Mendell, M.D. of Nationwide Children's Hospital, will present the data in an oral presentation of the abstract titled, "Results at 48 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne muscular dystrophy (DMD)."Dr. Mendell will present tomorrow from 2:30 to 4:00 p.m. WST UTC +8 hours/2:30 to 4:00 a.m. EDT. The presentation will describe new and previously reported efficacy and safety data from the Phase IIb study examining 48 weeks of treatment with eteplirsen in boys with DMD.Results from the Phase IIb extension study confirmed that treatment with Sarepta's lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort. Additional data to be presented includes: Dr. Mendell's presentation will be posted … Continue reading →

DART Therapeutics CEO Gene Williams says the company is competing against DMD, a fatal disease for which there is no treatment. Wednesday, October 10, 2012 DART Therapeutics LLC, a biotech focused on finding therapies for Duchenne Muscular Dystrophy (DMD), a progressive and fatal neuromuscular disease for which there is no current treatment, has completed tests on a new biomarker intended to speed drug development by cutting the number of patients needed for new drug trials by half. Cambridge-based DART conducted the trials from February through August of this year on 61 boys with DMD and 31 without. Newly appointed CEO Gene Williams told Mass High Tech that the biomarker - a device which measures a muscles response to a tiny electrical impulse - provides a more exact way to measure a potential drugs affects than the current standard for muscular diseases, which is to measure how far a patient can walk in six minutes. The so-called six-minute walk measurement can vary widely among boys, who might walk faster or slower depending on mood, said Williams. That means trials on potential DMD drugs must be large enough to factor out such variables, and as a result, many companies never test promising … Continue reading →

DART Therapeutics CEO Gene Williams says the company is competing against DMD, a fatal disease for which there is no treatment. Wednesday, October 10, 2012 DART Therapeutics LLC, a biotech focused on finding therapies for Duchenne Muscular Dystrophy (DMD), a progressive and fatal neuromuscular disease for which there is no current treatment, has completed tests on a new biomarker intended to speed drug development by cutting the number of patients needed for new drug trials by half. Cambridge-based DART conducted the trials from February through August of this year on 61 boys with DMD and 31 without. Newly appointed CEO Gene Williams told Mass High Tech that the biomarker - a device which measures a muscles response to a tiny electrical impulse - provides a more exact way to measure a potential drugs affects than the current standard for muscular diseases, which is to measure how far a patient can walk in six minutes. The so-called six-minute walk measurement can vary widely among boys, who might walk faster or slower depending on mood, said Williams. That means trials on potential DMD drugs must be large enough to factor out such variables, and as a result, many companies never test promising … Continue reading →

CAMBRIDGE, Mass., Oct. 10, 2012 /PRNewswire/ -- DART Therapeutics, LLC, an innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD), announced today the successful completion of a study to evaluate a promising new biomarker for DMD. The biomarker, electrical impedance myography (EIM), is a non-invasive and simple-to-use technique that allows the operator to measure the health of a muscle and track its changes over time. The information can help researchers evaluate how well a treatment is working to halt disease progression, which could support more precise drug therapy for DMD boys and faster, less expensive drug development for the disease overall. DMD is a progressive and fatal neuromuscular disease for which there is no effective treatment. Current drug development efforts rely on patient outcomes measurements that are either invasive and poorly tolerated or highly subjective and prone to variability. This can have a negative impact on the value of the studies, which must generate results that can be measured and reproduced over time. EIM is based on the observation that as a muscle becomes progressively diseased, an applied weak electrical current moves through it differently. The technique could provide drug developers with a highly precise way … Continue reading →

01-10-2012 06:54 Introduction of Ayurvedic Panchakarma by Dr Ravi Prasad (Head Of the Department ,PG Panchakarma, BRKR Ayurvedic College, Hyderabad) See the article here: Ayurvedic Panchakarma - Introduction - Treatment for Muscular Dystrophy - Video … Continue reading →