Muscular Dystrophy News -- ScienceDaily

Muscular Dystrophy News -- ScienceDaily
  • Origin of heart dysfunctions in myotonic dystrophy identified
    An international team of scientists is lifting the veil on the molecular mechanisms causing heart dysfunctions in myotonic dystrophy, a genetic disease affecting one person in 8,000.
  • Scientists reveal how osteopontin ablation ameliorates muscular dystrophy
    Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a new paper. The study adds support to the idea that osteopontin inhibitors could be used to treat patients with Duchenne muscular dystrophy.
  • Face- and eye-muscle research sheds new light on Duchenne muscular dystrophy
    Researchers have investigated the biochemical and physiological characteristics of orbicularis oculi, a group of facial muscles that control the eyelids and are selectively spared or involved in different neuromuscular disorders. What they found also helps to explain why another set of muscles -- the extraocular muscles that control the movement of the eye -- are not affected by Duchenne muscular dystrophy, congenital muscular dystrophy, and aging.
  • Decoding sugar molecules offers new key for combating muscular dystrophy
    Scientists have succeeded in decoding a sugar molecule and clarifying a mechanism linked to muscular dystrophy. Their discovery has potential implications for muscular dystrophy treatment.
  • New therapeutic target for spinal muscular atrophy
    Neuroscientists have discovered a specific enzyme that plays a critical role in spinal muscular atrophy, and that suppressing this enzyme's activity, could markedly reduce the disease's severity and improve patients' lifestyles.
  • Teaching stem cells to build muscle
    Researchers have identified pecific ways in which fetal muscle stem cells remodel their environment to support their enhanced capacity for regeneration, which could lead to targets for therapies to improve adult stem cells' ability to replace injured or degenerated muscle.
  • Stem cell gene therapy could be key to treating Duchenne muscular dystrophy
    Scientists have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. The stem cell gene therapy could be applicable for 60 percent of people with Duchenne, which affects approximately 1 in 5,000 boys in the US and is the most common fatal childhood genetic disease.
  • Robotic arm developed for Duchenne patients
    Researchers have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. They recently put the final touches to the first prototype for a discreet, body-connected support aid that can be worn under the clothing and that can support independent operation of the arm during important daily activities. Duchenne Muscular Dystrophy occurs in approximately 1 in 5000 live born boys. The muscles of Duchenne Muscular Dystrophy patients become weaker during their life. As a result, boys with Duchenne lose the ability to use their arms until, eventually, they are unable to use them at all.
  • Cancer drug shows promise for treating Duchenne muscular dystrophy
    A drug commonly used to treat leukemia is showing potential as a treatment that could slow the progression of the muscle-wasting condition, Duchenne muscular dystrophy.
  • Gene-editing technique successfully stops progression of Duchenne muscular dystrophy
    Using a new gene-editing technique, a team of scientists has stopped progression of Duchenne muscular dystrophy in young mice. DMD, the most common and severe form of muscular dystrophy among boys, is characterized by progressive muscle degeneration and weakness. The disease affects one in 3,500 to 5,000 boys, according to the Centers for Disease Control and Prevention and other estimates, and often leads to premature death by the early 30s.
  • Cancer drug shows promise in reducing toxic genetic material in myotonic dystrophy
    A drug used to treat cancer can neutralize the toxic RNA that causes the prolonged muscle contractions and other symptoms of myotonic dystrophy type 1, the most common form of adult-onset muscular dystrophy, a group of researchers has shown for the first time in cells and in a mouse model.
  • Critical pathway to improve muscle repair identified
    Researchers have identified the role of TNF receptor-associated factor 6 (TRAF6), an adaptor protein and E3 ubiquitin ligase, in ensuring the vitality of stem cells that regenerate muscle tissue.
  • Patient's priorities in treating rare muscular dystrophy
    A new study of individuals with myotonic dystrophy type 2 -- a rare form of muscular dystrophy -- has helped pinpoint the symptoms of the disease that are most important to patients. These findings could help create a roadmap for physicians to prioritize treatment of this complex, multi-system disease.
  • Duchenne muscular dystrophy is a stem cell disease
    For nearly 20 years, scientists have thought that the muscle weakness observed in patients with Duchenne muscular dystrophy is primarily due to problems in their muscle fibers, but new research shows that it is also due to intrinsic defects in muscle stem cells. Muscle stem cells that lack the dystrophin gene can't sense their orientation and produce ten-fold fewer muscle precursor cells, which in-turn generate fewer functional muscle fibers.
  • Target gene identified for therapies to combat muscular dystrophy
    Researchers have shown that a gene called Jagged1, or JAG1 for short, could be a target for the development of new approaches to treat Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration.