“This is the moment when a child who couldn’t even move his own body until two years ago stands on his own two feet.”
Mr. A still cannot forget the emotion of that day. To others, it is just a picture of a child standing, but just two years ago, all the child could do was lie down. The diagnosis is spinal muscular atrophy ( SMA ). About 20 patients suffer from this disease every year.
Of course, there is a cure. However, due to its high price of about 2 billion won, it was just a piece of cake for ordinary families. What lifted the parents’ broken hearts was the fact that Zolgensma, a gene therapy for SMA , was recognized by health insurance. And so far, Zolgensma has been administered to 15 patients.
According to Pusan National University Yangsan Hospital, Zolgensma from global pharmaceutical company Novartis was received at the hospital and administered to the 15th SMA patient.
SMA is a degenerative disease in which motor neurons in the spinal cord gradually degenerate and disappear, gradually weakening muscles throughout the body. It occurs at any time, including before birth, adolescence, and adulthood, but the younger the age of onset, the higher the severity and the shorter life expectancy.
Zolgensma was a dream for parents of SMA patients, including Mr. A. Of course, it is not possible to completely restore스포츠토토 the already damaged anterior horn cells of the spine, but by replacing the gene deficiency that causes the disease with a normal gene, it helps produce the proteins necessary to maintain nerve function. The biggest advantage is that the progression of SMA
can be prevented with just a once-in-a-lifetime medication . However, the price of over 2 billion won per shot was an obstacle that could not be overcome. However, the situation changed when Zolgensma was recognized as health insurance in August of last year. Patients who meet the health insurance benefit standards only need to pay the maximum out-of-pocket amount of 870,000 to 7.8 million won, depending on their income. At least there is no more giving up children for money.
The same was true for the 15th patient who visited Pusan National University Yangsan Hospital. Mr. B was unable to raise his legs from around 3 to 4 months old, and later showed developmental delays such as being unable to sit up or turn over. Eventually, he was diagnosed with SMA, and Zolgensma was administered on September 19, one month later.
Professor Kong Gong-hyeon of Pusan National University Yangsan Hospital said, “One-shot gene therapy provides a light of hope to patients with rare genetic diseases,” adding, “It brings great joy to patients with rare genetic diseases and at the same time brings innovative developments in the medical field, saving lives.” “I feel grateful to be able to spread hope,” he said.