Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it will receive an Innovation Credit of up to EUR 4 million from the Dutch government to support the development of AMT's gene therapy treatment for Duchenne Muscular Dystrophy (DMD).
Humans and mice have previously unknown and potentially critical differences in one of the genes responsible for Duchenne muscular dystrophy (DMD). Researchers writing in the open access journal BMC Biology have found that two major features of a key DMD gene are present in most mammals, including humans, but are specifically absent in mice and rats, calling into question the use of the mouse as the principal model animal for studying DMD.
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it has successfully treated Duchenne muscular dystrophy (DMD) in an animal model with its proprietary gene therapy. The proof of concept studies were performed in collaboration with the group of Professor Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated effectiveness in the heart as well as in skeletal muscles.
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
UCLA scientists have identified two chemicals that convince cells to ignore premature signals to stop producing important proteins. Published in the Sept. 28 edition of the Journal of Experimental Medicine, the findings could lead to new medications for genetic diseases, such as cancer and muscular dystrophy, that are sparked by missing proteins.
Galapagos NV (Euronext: GLPG), Charley's Fund Inc. and the Nash Avery Foundation announced today that they will collaborate to investigate the potential effectiveness of Galapagos' SARM candidate drug, G100192, in treating Duchenne muscular dystrophy. G100192 is an orally-available small molecule therapeutic, which has demonstrated successful Proof of Concept in pre-clinical studies for cachexia (the loss of weight and muscle mass).
AVI BioPharma, Inc., a developer of RNA-based drugs, today announced that Hong Moulton, Ph.D., Director of Discovery Research, will give oral presentations highlighting improved analogues of AVI's phosphorodiamidate morpholino oligomer (PMO) chemistry at two upcoming scientific meetings.
Researchers at the University of Minnesota and National Institutes of Health have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy.
Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.
Inflammation could contribute to bone loss in Duchenne's muscular dystrophy (DMD), a discovery made by a group of Italian researchers. Dr Anna Rufo and her colleagues found that levels of an inflammatory molecule, known as IL-6, are high in patients with DMD.
Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse.
Using a novel genetic technology that covers up genetic errors, researchers funded in part by the National Institutes of Health have developed a successful treatment for dogs with the canine version of Duchenne muscular dystrophy, a paralyzing, and ultimately fatal, muscle disease.
A University of Colorado at Boulder research team has identified a type of skeletal muscle stem cell that contributes to the repair of damaged muscles in mice, which could have important implications in the treatment of injured, diseased or aging muscle tissue in humans, including the ravages of muscular dystrophy.
A study by National Institutes of Health (NIH) researchers has revealed surprising new insights into the process used to initially identify an experimental drug now being tested in people with cystic fibrosis and muscular dystrophy.
A protein that was first identified for playing a key role in regulating normal heart rhythms also appears to be significant in helping muscle cells survive the forces of muscle contraction.
The overlooked and undervalued protein, sarcospan, just got its moment in the spotlight.
A medical research team at Carolinas Medical Center in Charlotte headed by Qi Long Lu, M.D., Ph.D., has made a discovery that holds promise to restore muscle function in patients afflicted by Duchenne muscular dystrophy (DMD).
By injecting purified stem cells isolated from adult skeletal muscle, researchers have shown they can restore healthy muscle and improve muscle function in mice with a form of muscular dystrophy. Those muscle-building stem cells were derived from a larger pool of so-called satellite cells that normally associate with mature muscle fibers and play a role in muscle growth and repair.
An investigational antiviral drug currently undergoing human trials in Europe for treating Hepatitis C infections may have potential to reduce muscle cell damage in Duchenne and other forms of muscular dystrophy (MD).