Let’s fight! The life-saving drug dropped from 700,000 to 33,000, and the first injection of SMA patients in Meizhou

The reporter learned from the Yuedong Hospital of the Third Affiliated Hospital of Sun Yat-sen University (hereinafter referred to as “Yuedong Hospital”) that on the morning of September 29, a patient with spinal muscular atrophy (hereinafter referred to as SMA) was diagnosed with spinal muscular atrophy (SMA). Chen Guoyi, an 11-year-old boy, successfully completed the first intrathecal injection of Nosinagen sodium in Yuedong Hospital.

SMA is a rare inherited neuromuscular disorder that affects approximately 1 in 10,000 people. The typical manifestations of SMA patients are muscle weakness, low muscle tone, muscle atrophy, and limited motor functions such as normal standing and walking.

“As a rare disease, SMA is currently the only treatment option in China is to use a life-saving drug – Nosinason Sodium Injection.” Rao Junping, Director and Chief Physician, Department of Neurology, Yuedong Hospital Said that this is the world’s first SMA precision targeted therapy drug, which was launched in China on April 28, 2019, and became China’s first drug that can treat SMA. On January 1 this year, Nosinagen Sodium Injection was officially included in the medical insurance, and it was reduced from 700,000 yuan to 33,000 yuan per injection.

“The life-saving medicine that used to be ‘highly expensive’ can now be affordable by ordinary families.” According to Rao Junping, after medical insurance reimbursement, patients actually only cost a few thousand yuan for an injection . “In addition, with the continuous improvement of the hospital’s treatment level, patients with this rare disease can receive good treatment at the hospital in front of their home, without having to go to big cities such as Guangzhou and Shenzhen, which is also a real relief for the public to see a doctor. “Rao Junping said.

It is worth mentioning that, before the operation, the hospital held a multidisciplinary consultation. On the basis of ensuring that the injection of Nosinagen sodium is safe, a follow-up plan was jointly developed. Comprehensive rehabilitation treatment.

Unlike most rare diseases, SMA is a rare disease with clinical heterogeneity, that is, although the etiology is the same, the clinical manifestations are different.

SMA is divided into five types: 0, Ⅰ, Ⅱ, Ⅲ and Ⅳ according to the age of onset and acquired motor function. Patients with SMA-0 type are the most ill, they develop the disease after birth and need to rely on respiratory support. Without treatment, most infants with SMA-I do not live to two years of age without respiratory intervention. Children with SMA-II, on the other hand, usually develop the disease within 6-18 months after birth, can sit alone, write, and can live to adulthood if they receive professional care and care.

Chen Guoyi was diagnosed with SMA-I when she was 16 months old. Since then, he has experienced breathing difficulties, and his lung capacity is only 1/5 that of a normal person. But by learning to sing, he continued to practice lung function, and miraculously took the stage to sing; and with the help of his father Chen Haiqiong, he practiced standing, calligraphy, etc.

Chen Haiqiong said that he began to pay attention to this drug a long time ago, but he could not afford it because of the high price. This injection, he hopes to improve his son’s physical condition.


Spinal muscular atrophy (SMA) is a rare inherited neuromuscular disease. The typical manifestations of SMA patients are muscle weakness, hypotonia, and muscle atrophy. Standing, walking and other motor functions are limited, and their motor development is significantly behind that of normal children, and they cannot even complete some of the most basic life-sustaining activities such as chewing, swallowing, and breathing, but their intellectual development is normal. The incidence of SMA is about 1/10,000, and the carrier rate of the disease-causing gene in the population is about 1/50.


Because of the severity of SMA, the high prevalence of the population, reliable and cost-effective detection methods, and the availability of genetic counseling and prenatal diagnosis, ACMG (American Academy of Medical Genetics and Genomics) in 2008 recommended that not only Couples with a family history of SMA should be screened for carriers, and the general population of all races and ethnicities should be screened for SMA carriers; the 2009 American College of Obstetricians and Gynecologists (ACOG) recommends that preconception and early pregnancy women should be screened for SMA carriers screening.

There are two suitable groups for carrier screening:

Family members of someone diagnosed with SMA, at-risk groups such as spouses of someone with SMA or a carrier, and the general population.

【All Media Reporter】Chen Zeming

【Correspondent】Zhong Lingfen Weng Taohua

【Author】 Chen Zeming

[Source] Southern Media Group Southern+Client