What Does Blocking Myostatin Do?
Blocking the protein known as myostatin, which limits muscle growth, has been under intense investigation as a strategy for the muscular dystrophies since 2002, when scientists found that mice with Duchenne muscular dystrophy (DMD) that were bred without myostatin were stronger and more muscular than their counterparts with normal myostatin levels.Read More
Through the Looking Glass with FSH Dystrophy Researchers
In 1990, Sara Winokur was a doctoral student in the laboratory of John Wasmuth, a professor of biological chemistry and a prominent researcher in genetics at the University of California at Irvine.It was an exciting time in genetics. The genes that, when mutated (flawed), cause diseases, were rapidly being identified. Among the first, in 1986, had been one for Duchenne muscular dystrophy.Read More
Scientists Bullish on Stem Cells for Muscle Repair
MDA grantee Giulio Cossu, director of the Stem Cell Research Institute of the San Raffaele Scientific Institute of Milan (Italy), was part of an Italian and French research team that restored mobility to two dogs and stabilized function in a third, using stem cells taken from muscle blood vessels.Maurilio Sampaolesi, at the San Raffaele Scientific Institute in Milan and the Institute of Myology at the University of Pavia (Italy), and colleagues, isolated mesoangioblasts from canine muscle biopsy samples and administered them through an artery into 10 dogs with a disorder resembling human Duchenne muscular dystrophy (DMD).Read More
Connecting the Dots
Just a few years ago, almost nothing could be said to parents like those in “Families Left with Questions,” other than that their child had a congenital (present at or near birth) form of muscular dystrophy.Andrew Loewi of Denver compares research on his daughter Samantha’s congenital MD (CMD) to Duchenne MD, for which the gene was identified in 1986. “What was so discouraging, so exasperating, for us, was that without even knowing what gene was involved, it seemed we were so far behind the eight ball that we’d never catch up.”Read More
Secrets of the Salamander: Can Stem Cells Repair Damaged Muscle Fibers?
Long before "stem cell" became a household term, people had observed that plants and animals can, within limits, repair damage they sustain.Wounds heal, broken bones knit, and lost blood is replenished. Mowed grass soon regrows, and barren trees sprout new leaves in spring. Cut off a limb of a salamander or some other amphibians, and it regrows. But the limbs of other animals, once gone, can’t grow back.Read More
Putting Out the Fire
These days, you can find Giovanna Albers at home with her family in Imperial, Mo., or on the job as a hostess at LongHorn Steakhouse in nearby Sunset Hills, or swimming laps at the YMCA.The trim, attractive, even athletic-looking 38-year-old radiates good health. So it comes as a surprise that her walk demonstrates profound weakness of her hip and thigh muscles, and that she occasionally falls.Read More
When Neuromuscular Disease Affects the Brain
If you’re the parent of a child with a neuromuscular disease, you’ve probably heard something about learning disabilities, mental retardation or emotional problems that accompany some of the muscular dystrophies and related diseases.But this information is often expressed in vague, general terms, leaving a parent wondering what specifically has gone wrong, whether the child’s school problem is directly related to his neuromuscular disease or not, and — perhaps most important — what can be done to help.Read More
Advances in Inclusion-Body Myositis
On winter mornings in icy Prescott, Wis., Ed Bankston gets up early, and, as he has done for many years, drives to his office in St. Paul, Minn. Bankston, 55, has been an attorney with the St. Paul District of the U.S. Army Corps of Engineers since 1988, and he now heads the district's legal department.Read More
The Brain in Duchenne Muscular Dystrophy
By the early 1990s, researchers began to see more than they had expected. The dystrophin gene wasn't just a muscle protein gene. There was also a brain form of dystrophin, made from the same gene, but in a slightly different way.Not surprisingly, scientists began to speculate that, if this brain protein were also flawed in Duchenne muscular dystrophy (DMD), it might account for the cognitive problems seen in the disease. And, if different people had different flaws in the same gene, it might account for some of the variation seen with these problems.Read More
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