New drug is said to help with muscle loss

New drug is said to help with muscle loss


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New therapy may delay the worsening of the course of the disease in patients with a certain form of muscle loss

Duchenne muscular dystrophy is the most common form of muscle wasting. “One in 3,500 boys developed Duchenne muscular dystrophy. The disease is usually diagnosed between the ages of two and four years. Loss of walking occurs at the age of eight to twelve years. Scoliosis occurs, the patient develops a breathing disorder, and in the end the heart muscle becomes ill, ”Günther Bernert, director of the Preyers Children's Hospital in Vienna, describes the disease in an interview with the news agency" APA ".

New drug for muscle loss is promising
Although the life expectancy of those affected has been significantly increased in recent decades, muscle wasting is not curable. "In the past, those affected were 15 years old, now we are close to 40 years old," reports the neuropediatrist at the annual conference for children's neurologists at the Austria Center Vienna.

Cortisone has long been considered the best way to delay the progression of the disease. There have been attempts in the field of gene therapy to replace the dystrophin gene which is defective in the disease, but have since been discontinued. A new drug that has been approved in Europe for about a year is now considered the most promising form of treatment. "Translarna" is a synthetic medicine that can delay the worsening of the disease in boys whose Duchenne muscular dystrophy is caused by a so-called nonsense mutation of the dystrophin gene.

“With these mutations, the reading process of the dystrophin gene is interrupted and no protein is produced. The active ingredient causes the genetic error to be skipped. At least an abbreviated protein is produced, ”explains Bernert. Therapy with “Translarna” is expensive, however. The treatment costs are currently around 300,000 euros per year.

Duchenne muscular dystrophy occurs very rarely
Duchenne muscular dystrophy is a rare genetic disease in which those affected gradually lose their muscle function. "Translarna" can only be used in a small group of Duchenne patients whose disease is based on a specific genetic defect. Affected patients suffer from a lack of normal dystrophin. It is a protein that protects the muscles from injury when contracting and relaxing. The absence of this protein causes the muscles to suffer permanent damage until they stop working. "Translarna" causes functioning dystrophin proteins to be formed again. (ag)

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