Iranian Firm Produces Special Drug for Treatment of SMA
“We succeeded in turning the company into a knowledge-based firm by developing a special drug product for SMA patients. This disease is a genetic disorder that causes nerve and muscle degeneration,” said Zahra Qassemi, an R&D expert at a pharmaceutical company.
She explained that a specific protein is not secreted in the body of these patients, which causes muscle degeneration, adding, “The drug helps this protein to be secreted again in the body and nerve and muscle function to improve significantly.”
“Although, according to the Swiss manufacturer, this drug is produced to prevent the progression of the disease, Iranian patients who have used the home-made product have also had a noticeable improvement in their movement,” Qassemi said.
“For instance, there was a child who was completely paralyzed before starting treatment, but after taking the drug he was able to sit up to a significant extent. This suggests that in addition to preventing the progression of the disease, some degree of improvement in muscle function is possible,” she noted.
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. It may also appear later in life and then have a milder course of the disease. The common feature is the progressive weakness of voluntary muscles, with the arm, leg, and respiratory muscles being affected first. Associated problems may include poor head control, difficulties swallowing, scoliosis, and joint contractures.
The age of onset and the severity of symptoms form the basis of the traditional classification of spinal muscular atrophy into several types.
Spinal muscular atrophy is due to an abnormality (mutation) in the SMN1 gene which encodes SMN, a protein necessary for the survival of motor neurons. Loss of these neurons in the spinal cord prevents signalling between the brain and skeletal muscles.
4155/v
