China News Service, Xining, March 1 (Li Jun) On the 1st, Yang Yang (pseudonym), a 7-year-old child with spinal muscular atrophy (SMA) in the Rehabilitation Department of Qinghai Women's and Children's Hospital, completed the injection of Nosinagen sodium. The condition was stable within 24 hours after the injection.
Yangyang is the first child in Qinghai Province to be injected with the drug Nosinagen Sodium Injection, a "targeted drug" for rare diseases, after it entered the Chinese medical insurance list.
February 28 is the 15th "International Rare Disease Day". The Rehabilitation Department of Qinghai Provincial Women's and Children's Hospital successfully completed the injection of Nosinason for Yangyang (pseudonym), a 7-year-old child with Spinal Muscular Atrophy (SMA). Sodium Injection.
The picture shows that Yangyang successfully completed the drug injection.
Photo by Qi Yuanping
Spinal muscular atrophy (SMA) is an autosomal recessive genetic disease caused by a gene defect. It often occurs in infants and young children. It is one of the most common genetic diseases that cause infant death and motor function degradation. With a population incidence of 1/6000 to 1/10000, it is the most common lethal genetic disease in infancy.
In May 2018, it was included in China's "First Batch of Rare Disease List".
Jin Hongfang, director of the Rehabilitation Department of Qinghai Women's and Children's Hospital, introduced that Nosinagen Sodium Injection is a drug for the treatment of spinal muscular atrophy, which was approved for the first time in the United States on December 23, 2016.
It is the world's first precision targeted therapy drug for SMA.
Administered by intrathecal injection, the drug can be delivered directly into the cerebrospinal fluid surrounding the spinal cord, thereby improving motor function, increasing survival, and altering the disease process of SMA.
On April 28, 2019, Nosinagen Sodium Injection was launched in China for the treatment of 5 types of SMA, and became the first drug in China to treat SMA.
"Yangyang began to suffer from poor motor development more than 6 months after birth, and his family took him to several hospitals. Finally, after genetic testing, he was diagnosed with SMA type II. At that time, there was no effective medicine for the disease in China. Walk." Jin Hongfang introduced that on December 3, 2021, the drug will be reduced from 700,000 yuan (RMB, the same below) per injection to 33,000 per injection, and Nosinagen Sodium Injection will be officially included in China's medical insurance.
The picture shows the doctor checking Yangyang's physical condition.
Photo by Qi Yuanping
After learning that the drug was included in the medical insurance catalog, Yangyang's parents came to the Rehabilitation Department of Qinghai Women's and Children's Hospital before the Spring Festival. .
On the 24th, Yangyang was admitted to the hospital for related examinations such as head MRI, coagulation function, liver and kidney function, and completed the motor function assessment on the 25th. At 11:40 am on the 28th, Yangyang received a lumbar puncture and intrathecal injection of Nosinasine sodium. Injection, the process went smoothly.
As of 11:40 on the 1st, Yangyang's condition was stable with no adverse reactions.
"Yangyang needs to inject 4 doses of Nosinagen sodium within 63 days, and then every 4 months, for life. He will receive follow-up medication and rehabilitation treatment in the hospital. Although the cost is expensive, Yangyang's family is subsistence allowance. For families, medical insurance can reimburse about 90% of the hospitalization expenses, which greatly relieves the heavy pressure brought by the disease to the families of children." Jin Hongfang said.
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