In the neurology ward of Hunan Children's Hospital, Wu Liwen, deputy director of the department and deputy chief physician, is doing muscle strength examination for fiber fiber.
Kang Qingyun (left), deputy chief physician of the Department of Neurology, and Liu Shulei (right), attending physician, injected Nocinasan sodium injection into a 4-year-old and 6-month-old girl with spinal muscular dystrophy.
Red Net Moment January 4 news (reporter Liu Dan correspondent Yao Jiaqi Xu Li) "Thanks to the good policies of the country, my children have hope!" On the morning of January 4, Ms. He from Xinhua County, Loudi City, rushed to Hunan Provincial Children's Hospital for prepaid diagnosis and treatment with her two-and-a-half-year-old daughter Fibrous Fiber (pseudonym) who suffered from spinal muscular atrophy (SMA for short). With the official implementation of the new version of the national medical insurance catalogue on January 1 this year, the original 700,000 injections of "sky-high" treatment drugs dropped to about 33,000 yuan, giving Ms. He's family a new hope, and fiber fiber also became one of the first children to receive injection treatment in the hospital.
"When the child was seven or eight months old, he couldn't climb, he thought it was just a little slower than others, and then he still couldn't do it when he was more than a year old, and he took it to the hospital for examination to find that it was this disease, and our whole family was confused, and we didn't know why this kind of thing happened to us." Ms. Ho's eyes lit up with tears. The daughter, who had just turned two and a half years old, was diagnosed with spinal muscular dystrophy type II at the age of one year and seven months. Because of the high price of drugs, he has been unable to treat his daughter after diagnosis. "Kids often say mom I want to walk, I want to go to kindergarten. But before I didn't dare to think about it, I was really excited to hear that the price reduction was really exciting, that is, I felt saved! There is hope! ”
At the beginning of December 2021, the National Medical Insurance Bureau announced 74 new drugs into medical insurance, and China's first imported drug approved for the treatment of spinal muscular dystrophy , Nosinasso Naoline Injection , suffered a "soul bargain", and finally entered the new version of the medical insurance drug catalog with a "floor price" of 33,000 yuan per needle. On the morning of January 4, 2022, nine children with spinal muscular dystrophy like fibrosis came to Hunan Children's Hospital to make an appointment for treatment, and they will also become the first batch of patients to receive injections after the drug is included in medical insurance.
Wu Liwen, deputy director and deputy chief physician of the Department of Neurology of Hunan Children's Hospital, introduced that spinal muscular atrophy, also known as SMA, is a type of disease caused by degeneration of motor neurons in the anterior horn of the spinal cord, resulting in muscle weakness and muscle atrophy. It is an autosomal recessive disease, and SMA is divided into 4 types from heavy to mild according to the age of onset and clinical course of the patient. As the disease progresses, muscle weakness can further lead to abnormalities in the skeletal, respiratory, digestive, and other systems, with respiratory failure being the most common cause of death.
In recent years, with the development of new therapeutic drugs and the improvement of treatment levels, more patients can achieve longer survival. Northinalsan Sodium Injection is the world's first precision-targeted SMA therapeutic drug. The drug is administered by intrathecal injection, which can directly deliver the drug to the cerebrospinal fluid around the spinal cord, thereby improving motor function, improving survival rates, and changing the disease course of SMA.
Wu Liwen said that the fiber can currently sit alone, but can not walk, there has been no regression of motor function, the overall situation is still relatively good. For this type of patient, after receiving the injection of Nocinasine sodium injection, it is hoped that the child will be able to stand up and walk slowly.
Due to the small number of people suffering from rare diseases and the limited market demand, rare disease drugs are also figuratively called "orphan drugs". In addition, even if there are drugs for rare diseases, the "sky-high price" of some rare disease drugs exceeds the ability of most people to pay, making patients discouraged and falling into a helpless situation. Making up for the shortcomings of rare disease patients has become an important part of the construction of China's medical insurance system, and it is also a hot spot in the national medical insurance negotiations every year.
"It's a subversive meaning for these children and families." Wu Liwen said that children with spinal muscular atrophy, if not treated with drugs, the quality of life will be worse and worse, and in the end the whole body can not move, including eating have difficulties. However, after medication, as long as it is not a complication, life expectancy will not have a great impact. And the motor function that the child has lost can be slowly acquired, so the value of this treatment is very huge.