Muscular Dystrophy News -- ScienceDaily

Muscular Dystrophy News -- ScienceDaily
  • 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.
  • Important regulation of cell invaginations discovered
    Lack of microinvaginations in the cell membrane, caveolae, can cause serious diseases such as lipodystrophy and muscular dystrophy. Researchers have now discovered a “main switch” that regulates the formation of these invaginations.
  • Muscle fibers grown in the lab offer new model for studying muscular dystrophy
    Investigators report that they have been able to drive cells to grow into muscle fibers, producing millimeter-long muscle fibers capable of contracting in a dish and multiplying in large numbers. This new method of producing muscle cells could offer a better model for studying muscle diseases, such as muscular dystrophy, and for testing out potential treatment options.
  • Inactivity reduces people's muscle strength
    It only takes two weeks of not using their legs for young people to lose a third of their muscular strength, leaving them on par with a person who is 40-50 years their senior, new research shows.
  • Cosmetic lip surgery may ease facial paralysis, small study suggests
    A cosmetic surgery that uses injections of hyaluronic acid to make lips appear fuller could also improve the lives of people with facial paralysis, according to results of a small study.
  • Gene discovery could lead to muscular dystrophy treatment
    Researchers have made a critical discovery about a gene involved in muscular dystrophy that could lead to future therapies for the currently untreatable disease. Facioscapulohumeral muscular dystrophy (FSHD) is a progressive wasting disease that affects the face, arms and shoulders. It is most commonly diagnosed in teenage or early adults, and though it is rarely fatal is it very debilitating.