【Subtitle】Recently, the rare disease life-saving drug Noxinagen Sodium Injection, which was used for nearly 700,000 yuan per injection, was included in the medical insurance.
[1] What disease does this sky-high-priced life-saving medicine cure?
[Concurrent] Lu Yan, Deputy Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
The 700,000 Noxinagen Sodium Injection treats spinal muscular atrophy (SMA), which is a rare disease. Its main clinical manifestations are weakness and atrophy of limbs. We are clinically different from the age of onset. Divided into about 5 types, the earlier the age of onset, the more severe the symptoms.
In one type, when the mother is pregnant, the child will find that the baby has little fetal movement, and after birth, there will be sucking weakness, respiratory muscle weakness, and soon die.
In addition, children who became ill at about six months of age cannot sit, and they may die within two years of age.
[2] What is the incidence of SMA?
[Concurrent] Lu Yan, Deputy Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
The incidence rate of this disease in foreign statistics is about 4 to 7 per 100,000, because our country has a large population base. Estimated in our country, the incidence rate of live births per year is 1,200 children per year, but because of its He died soon, so the actual stock of patients may currently be estimated to be about 30,000.
[3] How to treat SMA?
[Concurrent] Lu Yan, Deputy Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
This disease is caused by genetic mutations. According to our common sense, we cannot change the gene itself for genetic mutation diseases. Therefore, there is basically no good treatment method, and all are symptomatic treatment.
Now there is such a medicine that can change the proteins produced by genes through this modification method, and then give the patient a treatment plan, so we say that it is a life-saving medicine.
[4] How many injections do I need to take the sky-high life-saving medicine?
[Concurrent] Lu Yan, Deputy Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
The drug is injected intrathecally. Generally, there is an intensive treatment in the first 8 weeks, with 4 injections 4 times, and after 8 weeks, a maintenance treatment every 4 months.
[5] What good news does the soaring price of life-saving medicine bring to patients?
[Concurrent] Lu Yan, Deputy Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
Although this medicine entered the Chinese market in 2019, because of its high price, few patients can afford it, and few hospitals can access this medicine, so there are very few hospitals that actually treat it.
Now it has entered medical insurance, and the price is relatively close to the people. I believe that as long as patients have demand, we will have more hospitals that can enter this medicine to serve our patients.
Reporter Wen Mengxin reports from Beijing
Editor in charge: [Liu Xian]