GlaxoSmithKline releases results of phase 1 exon skipping trial
The drug company GlaxoSmithKline has today announced results of a phase 1 clinical trial of a potential exon skipping drug -now called drisapersen - in boys with Duchenne muscular dystrophy who are unable to walk. This drug, a molecular patch for exon 51 of the dystrophin gene, has previously been tested in boys with Duchenne muscular dystrophy who are still able to walk and has generated encouraging results. The main aim of this trial was to make sure there are no additional safety issues for wheelchair users and to test whether different doses of the potential exon skipping drug were safe and well tolerated.
The company recruited 20 boys who were given one of three doses of drisapersen or a placebo (an inactive substance). The participants received a single injection of the drug and were monitored for up to 6 months. All the boys who received the potential drug experienced some mild side effects such as redness or swelling of the injection site and all the boys given the highest dose of the potential drug experienced fever. However, the researchers say no serious side effects were observed.
The pelvic floor is made up of muscles, fascia and other tissues. These form a sling from the pubic bone to the tailbone. Pelvic floor muscle assist in maintaining upright posture, supporting abdominal and pelvic organs, and help to control bladder, bowel and sexual activity. Pelvic floor dysfunction occurs when these muscles become weak, overused, overactive causing pain, weakness in controlling bowel and bladder symptoms, reduced sexual function. There is often related impairments of the sacroiliac joint, low back, coccyx and/or hip joint, which accompany this condition, and can contribute to pain and loss of function.
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