Muscular Dystrophy

Life expectancy is lower for blacks compared with whites in the United states by about five years, mostly because of more heart disease among blacks. But researchers reported Monday on a shocking disparity in death rates among blacks with muscular dystrophy that is likely due to inequality in healthcare. Muscular dystrophy is an incurable muscle disease that often leads to death in early adulthood due to respiratory or cardiac failure. The study reported Monday in the journal Neurology examined 18,315 deaths associated with muscular dystrophy in the United States over a 20-year period and found that African Americans with the disease die 10 to 12 years before their white counterparts -- a healthcare disparity gap "that is among the largest ever demonstrated," said the authors of an editorial accompanying the study.

For the first time in 45 years, Jerry Lewis will not be pleading for donations in front of a camera Labor Day weekend after he was abruptly dismissed as the host of the Muscular Dystrophy Assn.'s telethon, an event that drew attention to the childhood disease and in its heyday was an annual television highlight. The group said the 85-year-old legendary comedian would not appear on this year's telethon, and would no longer serve as its national chairman, a position he held for nearly 60 years.

Boys with Duchenne muscular dystrophy, the most common type of muscular dystrophy, who are treated with long-term steroid therapy are able to walk on their own for several more years than boys not treated with steroids, according to findings published in the May issue of Neurology. However, this benefit comes at the cost of more fractures.

Loss of muscle mass is a fact of life starting in middle age -- we lose about 1% a year in a phenomenon called sarcopenia. Researchers say they've not only discovered the cause of that loss but may have found a drug that could help it as well. The online study, released Tuesday in the journal Cell Metabolism , maintains that sarcopenia happens because of calcium seepage from the ryanodine receptor channel complex, a group of proteins found in muscle cells. A domino effect follows: The leaks kick off a chain reaction resulting in muscle fibers not being able to fully contract.

City firefighters will be collecting donations outside Disneyland on Saturday for the Muscular Dystrophy Assn. Calling it Fill the Boot Day, the firefighters will stand at the corner of Harbor Boulevard and Katella Avenue from 9 a.m. to 4 p.m. They will ask drivers and others to put money into a rubber fire boot. The donations collected in Anaheim will be forwarded to the International Assn. of Firefighters, which is organizing Fill the Boot Days nationwide.

The Muscular Dystrophy Assn.'s third annual John Zimmerman Memorial Dinner Auction will be held Aug. 1 at the Anaheim Hilton and Towers. Funds raised at the dinner go toward Muscular Dystrophy Assn. research and patient-services programs. Tickets are $65 per seat and $650 per table. The latter includes eight seats and two complementary seats donated back to Marine Corps guests.

Researchers appear to have partially corrected muscular dystrophy in mice by injecting corrective genes directly into the mice's muscles, according to a report last week in the British journal Nature. The study points the way toward the eventual use of gene therapy to arrest or reverse muscular dystrophy in patients, but years of research will be required before then, according to researchers from the University of Wisconsin and the John Radcliffe Hospital in Oxford, England.

Important evidence that human stem cells can be implanted and become part of muscles suggests that muscular dystrophy may yet become treatable, scientists announced. The discovery--made because of an odd biomedical coincidence--showed new cells can infiltrate a person's damaged muscles, take root and survive for years. This is what is needed, on a larger scale, to attack the inherited disease. "This says that we can get those cells recruited" into weakening muscles, said Dr.

Researchers believe that within three months they will isolate the entire gene responsible for Duchenne muscular dystrophy, which would be a major step toward unraveling the mystery of the fatal disease that strikes one in every 3,300 males. Last fall, researchers at Boston Children's Hospital announced that they had identified the gene that causes the most common form of muscular dystrophy and isolated part of it.

A Brazilian scientist has found a drug that appears to slow the deterioration that typifies Duchenne muscular dystrophy, an invariably fatal disease that afflicts about 20,000 boys in the United States at any one time. However, there is a catch: The drug has the unwanted effect of stunting the growth of children who receive it.

A genetic technique that allows the body to work around a crucial mutation that causes Duchenne muscular dystrophy increased the mass and function of muscles in a small group of patients with the devastating disease, paving the way for larger clinical trials of the drug. The study in a handful of boys age 5 to 15 showed that patients receiving the highest level of the drug, called AVI-4658 or eteplirsen, had a significant increase in production of a missing protein and increases in muscle fibers.

Life expectancy is lower for blacks compared with whites in the United states by about five years, mostly because of more heart disease among blacks. But researchers reported Monday on a shocking disparity in death rates among blacks with muscular dystrophy that is likely due to inequality in healthcare. Muscular dystrophy is an incurable muscle disease that often leads to death in early adulthood due to respiratory or cardiac failure. The study reported Monday in the journal Neurology examined 18,315 deaths associated with muscular dystrophy in the United States over a 20-year period and found that African Americans with the disease die 10 to 12 years before their white counterparts -- a healthcare disparity gap "that is among the largest ever demonstrated," said the authors of an editorial accompanying the study.

On a recent afternoon, comedy legend Jerry Lewis cracked open a diet soda and dimmed the lights inside a casino ballroom to drink in the spectacle of Charlie Chaplin impersonating Hitler. A scene from Chaplin's 1940 Nazi satire "The Great Dictator" flickered across a bank of monitors, part of a video montage Lewis was editing together for his signature cause, the Muscular Dystrophy Assn. Telethon . The 211/2 -hour annual event has raised $2.45 billion for "Jerry's kids" to date; its 45th edition kicks off on some 170 television stations Sunday evening.

Mice are used throughout medical research to provide models for human disease. The more accurate the mouse model of the disease, the better for scientists trying to understand disease processes and treatments. Researchers this week announced they have developed what they say is a more realistic mouse model for Duchenne muscular dystrophy. In the new model, scientists can study muscle damage that is similar to what occurs in humans with the disease. The model produces human-like glycosylation, the process by which human cells coat themselves with long sugar chains that are attached to proteins.

Rarely in life do the personal and professional become the bittersweet blend that Stan Nelson and Carrie Miceli experience every day. At work, the medical researchers at UCLA painstakingly delve into the genetic underpinnings of Duchenne muscular dystrophy, the most common of childhood's lethal DNA-linked diseases. At home, they treasure their family time with sons Calvin, 16, and Dylan, 9. Such time is precious. Dylan was diagnosed with Duchenne as a toddler. Miceli and Nelson don't dream of a cure for their youngest son. Heavily schooled in objectivity and neutrality, they are able to tolerate the often-creeping pace of science and yet savor its progress.

Jerry Lewis is known as one of show business' silliest and most outrageous clowns. But when he was honored Sunday night during the Oscars for his humanitarian work, he played it straight. "This touches my heart and the very depths of my soul, not only because of who this award is from, but who it will benefit," Lewis said in accepting the Jean Hersholt Humanitarian Award. "My humility is staggering."

For the second time in three weeks, researchers have reported promising results in humans with a proposed new genetic therapy for Duchenne muscular dystrophy. At a Wednesday press conference, a Canadian researcher said that the unusual therapy has been found to be both safe and feasible in tests on three young boys, but that it is too early to tell if the treatment is effective in reversing the course of the disease.

A small safety trial has suggested that a new type of therapy for Duchenne muscular dystrophy may partially replace a missing protein and -- at the very least -- slow the course of the disease, Dutch researchers reported Thursday in the New England Journal of Medicine. The experimental drug, developed by the Dutch pharmaceutical company Prosensa, is an "antisense" fragment of ribonucleic acid (RNA) that can mitigate the effects of a genetic mutation.

Stem cells taken from Duchenne muscular dystrophy patients and genetically modified eased symptoms of the disease in mice, strengthening their muscles and allowing them to run longer on a treadmill, researchers reported Wednesday in the journal Cell Stem Cell. The patients have a nonfunctional form of a protein called dystrophin. Researchers modified the muscle cells from the patients to produce a shortened but still effective form of dystrophin.