Research
Research Updates Summer 2009
Story includes research about: Becker muscular dystrophy, Duchenne muscular dystrophy and oculopharyngeal muscular dystrophy. Scientists at Children’s National Medical Center in Washington, Carolinas Medical Center in Charlotte, N.C., and the National Center of Neurology and Psychiatry in Tokyo, have successfully treated dogs with a disease closely resembling Duchenne muscular dystrophy (DMD), using a molecular treatment strategy called exon skipping.
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ALS: A Vicious Cycle
A vicious cycle in which damage to nerve cells (neurons) in the spinal cord results in the loss of an important mechanism to protect neurons, causing more neuron loss, has been identified as a possible contributor to ALS (amyotrophic lateral sclerosis). Identification of this pathway opens the door to targeting it with therapeutic agents.
Read MoreSMA Research
This decade, for the first time, scientific research in spinal muscular atrophy (SMA) has yielded enough information to allow the pursuit of several avenues of disease treatment and prevention.Most are centered around increasing cellular levels of a protein called SMN (survival of motor neuron), first identified in the mid-1990s as the root cause of almost all cases of SMA.
Read MoreResearch Updates Winter 2009
Story includes research items about: centronuclear myopathy; Duchenne, limb-girdle, and Emery-Dreifuss muscular dystrophies; and spinal muscular atrophy.Missing or reduced levels of an enzyme known as neuronal nitric oxide synthase (nNOS) at its normal location on muscle-fiber membranes prevents blood vessels that supply active muscles from relaxing normally, leading to exercise-associated fatigue, says a team of researchers from the University of Iowa in Iowa City, the University of Michigan in Ann Arbor, and the University of Washington School of Medicine in Seattle.
Read MoreThe Genie’s Out of the Bottle
From the Human Genome Project to TV medical and crime shows, to an evergrowing list of genetic tests purported to diagnose diseases, trace your ancestry or predict your future, DNA seems to be the subject of the day.Even those who choose not to undergo testing find it hard to ignore. Whether or not to get tested is a personal decision. But since the DNA testing genie isn’t going back in the bottle, it’s a good idea to get to know him.
Read MoreEuropean Team Combines Stem Cells, Gene Correction to Treat DMD-Affected Mice
A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support.
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Taking a Closer Look at Myotubular Myopathy
When Patrick and Sarah Foye of Pine Brook, N.J., had their baby boy, Adam, in 2001, there was little reason to believe at first that anything was wrong. "Adam had a normal birth," Sarah says, but things didn't go as well as expected in the months that followed.Patrick is a physician specializing in rehabilitation, and Sarah is an occupational therapist, so they were quick to note that something wasn’t right, even though Adam was their first child.
Read MoreResearch Updates September-October 2007
Diseased featured in this article include: Duchenne muscular dystrophy, Friedreich's ataxia and myotonic musuclar dystrophy.MDA-supported researchers at the University of Pennsylvania and the University of Ottawa report finding a molecule that keeps the muscle protein known as utrophin confined to one small area of a muscle fiber and thereby limits its potential as a therapy for Duchenne muscular dystrophy (DMD). Releasing this brake by blocking this molecule could become a strategy for treating the disease.
Read MoreTruth, Lies and Tracheostomies
When neuromuscular disease weakens the muscles used for breathing, many people benefit greatly from noninvasive ventilation (NIV), which can add years of breathing support.But when a tracheostomy and ventilator are suggested for better breathing, some people see NIV as “enough” and a trach and vent as somehow “too much.”
Read MoreWhat 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.
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