Muscular Dystrophy News -- ScienceDaily

Muscular Dystrophy News -- ScienceDaily
  • 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.
  • Manipulating cell signaling for better muscle function in muscular dystrophy
    Researchers report on their discovery of a way to bypass faculty cell signalling that leads to muscle damage in Duchene muscular dystrophy. This work suggests a new therapeutic strategy for patients with Duchene muscular dystrophy, a progressive neuromuscular condition, caused by a lack of dystrophin, that usually leaves patients unable to walk on their own by age 10-15.
  • Gene therapy treats all muscles in the body in muscular dystrophy dogs
    Muscular dystrophy, which affects approximately 250,000 people in the US, occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of researchers has successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.
  • Mouse model of Duchenne muscular dystropy identifies potential new approaches to therapy
    Genetic ablation of P2RX7 can improve muscle function and partially correct cognitive impairment and bone loss in a mouse model of Duchenne muscular dystrophy, according to a new study. The research additionally suggests that P2RX7 antagonists can reduce certain DMD phenotypes in these mice.
  • New research sees zebrafish earn their stripes in the fight against Muscular Dystrophy
    New research has demonstrated a new method for observing the behavior of the protein Dystrophin in a living animal cell, in real-time. This breakthrough may provide a key to understanding how to treat the genetic disease, Muscular Dystrophy.
  • RNA editing technique treats severe form of muscular dystrophy
    An RNA editing technique called 'exon skipping' has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment. The discovery stems from the persistence of a father, whose two sons were diagnosed with a rare and severe form of muscular dystrophy, and his search for and partnership with the genetic scientist who studies the disease. The therapy is being developed with the goal of clinical trials.
  • Human muscle stem cells isolated
    Researchers have successfully isolated human muscle stem cells and shown that the cells could robustly replicate and repair damaged muscles when grafted onto an injured site.
  • iPS cells discover drug target for muscle disease
    Duchenne muscular dystrophy (DMD) is disease that leads to the degeneration of muscle due to dysfunctional expression of the protein dystrophin. A new iPS cell model found that the early stages of DMD development can be attributed to a specific family of ion channels that allows an abnormal influx of calcium to enter the cell. This same model can be used for drug discovery to correct the channel behavior, experts say.