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Alan Jackson got choked up on stage.May 17 marked Alan Jackson's last stop on his current tour — which is going to be his last. Y'all may have heard that I'm kinda winding down, and in fact this is my last road show of my career," Jackson, 66, said in front of a roaring Milwaukee crowd. "Y'all gonna make me tear up out here. But I will say, this is my last road show out here, but we're planning on doing a big finale show in Nashville next summer sometime. It just felt like I had to end it all...

For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy. On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year. That decision, documents concurrently released by the agency show, was made almost exclusively by Peter Marks, the agency’s director of the Center for Biologics Evaluation and Research. He...

Researchers have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy. The researchers found that the drug—known as a colony-stimulating factor 1 receptor (CSF1R) inhibitor—helped slow the progress of Duchenne muscular dystrophy in mice by increasing the resiliency of muscle fibers. "This is a class of drug that is already being used in clinical trials to treat rare forms of cancer," says Dr. Farshad Babaeijandaghi. Duchenne muscular dystrophy (DMD) is a severe genetic disorder that leads to progressive muscle weakness and degeneration. "While this is not a cure, it could significantly delay disease progression,...

CAMBRIDGE, Mass. — When Sharif Tabebordbar was born in 1986, his father, Jafar, was 32 and already had symptoms of a muscle wasting disease. The mysterious illness would come to define Sharif’s life. Jafar Tabebordbar could walk when he was in his 30s but stumbled and often lost his balance. Then he lost his ability to drive. When he was 50, he could use his hands. Now he has to support one hand with another. No one could answer the question plaguing Sharif and his younger brother, Shayan: What was this disease? And would they develop it the way their...

Results of a compassionate-use study released in STEM CELLS Translational Medicine show promising results for treating muscular dystrophies with mesenchymal stem cells (MSCs) derived from Wharton’s jelly (WJ), a substance found in the umbilical cord. Led by doctors at Klara Medical Center (KMC), Czestochowa, Poland, these WJ-MSC treatments resulted in significant improvement in several body muscles in most of the patients, with no serious side effects. “Administration of WJ-MSCs in neurological indications is controversial; still, this paper shows that cell therapy is a reasonable experimental treatment option, although the eligibility criteria for treatment needs to be optimized,” said Beata Świątkowska-Flis,...

Dietary intervention could benefit heart health in those with muscular dystrophy. That's according to new research published in Experimental Physiology. If these findings are confirmed in humans, it could mean that off the shelf supplements could improve health and life expectancy. Scientists from Iowa State University, Auburn University and the University of Montana in the United States found that supplementing the mice's food with quercetin (a flavonol found in many fruits, vegetables, leaves, and grains) improved biomedical outcomes, providing an inflammatory and antioxidant effect. To the groups' surprise, they also found that the quercetin-fed mice were more active than the...

Duchenne muscular dystrophy is caused by mutations that disrupt the normal function of a gene on the X chromosome. The disease mostly affects boys – about one in 3,500 – because they have only one X chromosome. Since girls have two X chromosomes they tend to have a working backup if one copy of the gene is damaged. As a result girls tend not to be affected, but can be carriers and may pass mutated genes on to their children. The key gene in muscular dystrophy is needed to make dystrophin, a protein which is crucial for strong muscle fibres....

The Muscular Dystrophy Association telethon, a Labor Day television tradition for decades, is ending. MDA said Friday that “the new realities of television viewing and philanthropic giving” make it the right time to end the annual event, memorably hosted for most of its life by Jerry Lewis. Celebrities including Frank Sinatra, John Lennon and Michael Jackson to Pitbull and Jennifer Lopez have performed on the telethon, first hosted by Lewis and Dean Martin in 1956. It moved to Labor Day in 1966. The telethon ran 21 and a half hours in 2010, Lewis’ last year as host, and had dwindled...

With the attempt to limit the deductibility of contributions to charities by high-earners, the Obama Administration has opened a multi-front attack against the American tradition of “neighbor helping neighbor” and financially successful people turning large portions of their wealth over to charities that improve the American culture in august manners. The governmental power grab competes with the attempts to control the financial sector for its significance and may exceed it in the long-term effect upon our culture. One first has to marvel at how liberals can twist logic to rationalize a tax increase. It happens regularly from the local government...

University of Illinois comparative biosciences professor Suzanne Berry-Miller, veterinary clinical medicine professor Robert O’Brien.Researchers have shown that transplanting stem cells derived from normal mouse blood vessels into the hearts of mice that model the pathology associated with Duchenne muscular dystrophy (DMD) prevents the decrease in heart function associated with DMD. Their findings appear in the journal Stem Cells Translational Medicine. Duchenne muscular dystrophy is a genetic disorder caused by a mutation in the gene for dystrophin, a protein that anchors muscle cells in place when they contract. Without dystrophin, muscle contractions tear cell membranes, leading to cell death. The lost...

LAS VEGAS, Sept. 5, 2011 /PRNewswire-USNewswire/ -- The new era of primetime Muscular Dystrophy Association Telethons has started. In six primetime hours last night, the organization leading the fight against progressive muscle diseases delivered on its promise of an outstanding 46th annual MDA Labor Day Telethon. MDA today announced that its 2011 Telethon raised $61,491,393 -- up from the $58,919,838 achieved during the prior year's 21.5-hour Telethon. This is the biggest Telethon achievement since 2008, when the global recession began. Contributions can still be made online at mda.org or by calling 800-FIGHTMD (800-344-4863). "We're so grateful to everyone who made...

Few TV traditions in America are as popular as the annual MDA Labor Day Telethon, but this year’s is still shrouded in mystery as to what happened between entertainment legend and longtime host Jerry Lewis and the Muscular Dystrophy Association. Not one mention of his name in the two latest MDA press releases about the telethon. That’s not only downright rude and disgraceful, but also a tragedy. This year’s show, broadcast live again from South Point on Sunday, has been cut to 6 hours from the long-standing 21-hour marathon. MDA says the new look will be more fast-paced and hopefully...

Jerry Lewis announced on Monday that he is retiring as the longtime emcee of the annual Muscular Dystrophy Association Labor Day Telethon. “As a labor of love, I’ve hosted the annual Telethon since 1966, and I’ll be making my final appearance on the show this year by performing my signature song, ‘You’ll Never Walk Alone’,” the legendary 85-year-old comedy entertainer said in a statement on the MDA website. “I’ll continue to serve MDA as its National Chairman – as I’ve done since the early 1950′s. I’ll never desert MDA and my kids.” According to the MDA, Lewis has logged more...

Trashing the muscular dystrophy telethon is like dissing Christmas. It ticks people off. Even if the offender is a former Jerry's Kid. Laura Hershey was born with a form of MD and spent her poster childhood appearing helpless at fundraisers. She became a celebrity in the '70s as the face of the condition in Colorado. She remembers being a prop in the TV studio that was broadcasting local parts of the telethon. "To whole families driving by to drop their contributions in a giant fishbowl outside the studio; to the camera's blinking red light; to the anchorman who squatted next...

The human genome is riddled with dead genes, fossils of a sort, dating back hundreds of thousands of years — the genome’s equivalent of an attic full of broken and useless junk. Some of those genes, surprised geneticists reported Thursday, can rise from the dead like zombies, waking up to cause one of the most common forms of muscular dystrophy. This is the first time, geneticists say, that they have seen a dead gene come back to life and cause a disease. “If we were thinking of a collection of the genome’s greatest hits, this would go on the list,”...

An experimental procedure that dramatically strengthens stem cells' ability to regenerate damaged tissue could offer new hope to sufferers of muscle-wasting diseases such as myopathy and muscular dystrophy, according to researchers from the University of New South Wales (UNSW). The world-first procedure has been successfully used to regrow muscles in a mouse model, but it could be applied to all tissue-based illnesses in humans such as in the liver, pancreas or brain, the researchers say. The research team, [...] adapted a technique currently being trialled in bone marrow transplantation. Adult stem cells are given a gene that makes them resistant...

Researchers at the Joslin Diabetes Center have demonstrated for the first time that transplanted muscle stem cells can both improve muscle function in animals with a form of muscular dystrophy and replenish the stem cell population for use in the repair of future muscle injuries. "I'm very excited about this," said lead author Amy J. Wagers, Ph.D., Principal Investigator in the Joslin Section on Developmental and Stem Cell Biology, principal faculty member at the Harvard Stem Cell Institute and Assistant Professor of Stem Cell and Regenerative Biology at Harvard University. "This study indicates the presence of renewing muscle stem cells...

Researchers at the Joslin Diabetes Center have demonstrated for the first time that transplanted muscle stem cells can both improve muscle function in animals with a form of muscular dystrophy and replenish the stem cell population for use in the repair of future muscle injuries. “I’m very excited about this,” said lead author Amy J. Wagers, Ph.D., Principal Investigator in the Joslin Section on Developmental and Stem Cell Biology, principal faculty member at the Harvard Stem Cell Institute and Assistant Professor of Stem Cell and Regenerative Biology at Harvard University. “This study indicates the presence of renewing muscle stem cells...

AP Science Writer Modified stem cells from muscular dystrophy patients eased symptoms of the disease in mice, says a small study that raises hopes for treating patients with tissue from their own bodies. The mice showed stronger muscles and ran longer on a treadmill than diseased mice that weren't treated. Other experimental treatments for muscular dystrophy have also produced encouraging results in lab animals, but experts said the new study shows promise for yet another approach. The paper focused on Duchenne muscular dystrophy, a muscle-wasting genetic disorder appearing in boys that occurs in about 1 in every 3,500 male births....

Showman Jerry Lewis sets a goal for his annual Labor Day Telethon to benefit the Muscular Dystrophy Association of $1 more than the previous year. He met that mark, plus almost $3 million on Monday. "We did it. We did it. I got my buck more. And more. We can go shopping," a jubilant Lewis said as the tote board topped $63.7 million for his 42nd telethon. Last year's record was $61 million. As in the past, the International Association of Fire Fighters anchored the donations from local fill-the-boot drives with $25.2 million, eclipsing last year's $23.5 million. "Whoa, whoa...