The Kessler Foundation and the New Jersey Institute of Technology (NJIT) will share a $5 million federal grant, titled Rehabilitation Engineering Research Center on Wearable Robots, to develop a wearable robotic exoskeleton for use in patients with Duchenne muscular dystrophy (DMD), as well as other applications for spinal cord injury and stroke.
Advances in stem cell therapy using patients’ own cells with genetically modified dystrophin have been hampered by the large size of the dystrophin gene, which are difficult to pack into a delivery system. Now, scientists at University College London have managed to produce a gene construct much bigger than was…
Akashi Therapeutics announced they are suspending both dosing and patient enrollment in the HALO trial – a study investigating their experimental therapy, HT-100, in patients with Duchenne muscular dystrophy. The trial’s suspension came after a patient enrolled in the highest dose-group of the study began experiencing life-threatening adverse effects. At this time,…
Duke University researcher Brent Hoffman is investigating the mechanical nature of major diseases — a research area named mechanobiology — including muscular dystrophy. Hoffman’s research could lead to the development of new treatment approaches for mechanosensitive diseases. Many complicated diseases have a little understood mechanical component. Dr. Hoffman, an assistant…
Researchers have found that RNA sequencing is able to identify disease-causing genetic mutations in non-coding regions of the DNA. The technique can be used to aid in diagnosing muscular dystrophy (MD). The study, “RNAseq analysis for the diagnosis of muscular dystrophy,“ appeared in the journal Annals of Clinical and Translational…
Researchers from Duke University have succeeded in treating an adult mouse model of Duchenne muscular dystrophy using the gene editing system CRISPR — the first successful treatment of a genetic disease in an adult animal utilizing a method that has the potential to be used in humans. In spite of recent advances…
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