In terms of SMA treatment drugs, Beijing Jingdu Children's Hospital keeps up with international frontiers and actively introduces effective therapeutic drugs. Among them, Nusinersen Injection, as the world's first approved SMA treatment drug, has attracted much attention. Encouragingly, after arduous negotiations between the National Medical Insurance Administration and enterprises, the price of this once "sky-high drug" has dropped significantly. In China, its price is only about one-twentieth of that in the United States. In the United States, a dose of Nusinersen Injection is priced as high as 125,000 USD, and the treatment cost in the first year is about 750,000. In China, after being included in medical insurance, the price per injection has dropped to approximately 4,633 USD. The patient's out-of-pocket expense in the first year is around 5,616. This huge price difference enables more Chinese families to afford the treatment cost, giving sick children the opportunity to receive standardized treatment.
In addition to Nusinersen Injection, the first oral SMA drug Risdiplam has also passed the national medical insurance negotiation, with its price dropping from 14,380 USD
per bottle to 530 USD, and the new medical insurance price was implemented on March 1, 2023. Beijing Jingdu Children's Hospital quickly applied these beneficial drugs to clinical treatment, bringing hope to the children.
Beijing Jingdu Children's Hospital has established a professional multidisciplinary diagnosis and treatment team to provide comprehensive medical services for children with SMA. Team members include neurology experts, pediatricians, rehabilitation therapists, nutritionists, and psychologists, etc.
Neurology experts, with rich experience, can accurately diagnose the condition of children with SMA and formulate personalized treatment plans according to different types and severity. Pediatricians closely monitor the children's growth and development during daily treatment, timely handle various complications caused by the disease, and ensure the children's physical health. Rehabilitation therapists are crucial for children with SMA. They will tailor rehabilitation training plans, including physical therapy and occupational therapy, according to the specific conditions of the children. Through long-term and systematic rehabilitation training, they help the children maintain muscle strength and joint mobility as much as possible, delay the process of muscle atrophy, and improve self-care ability. For example, through specific muscle exercises and joint activity training, some children with mild SMA can maintain good limb movement function and achieve independent walking.
Nutritionists will formulate scientific and reasonable diet plans for the children to ensure they get sufficient and balanced nutrition to support physical growth and development and cope with disease consumption. Since children with SMA may have problems such as dysphagia, nutritionists will adjust the texture of food and feeding methods according to the actual situation to ensure the children's nutritional intake. Psychologists pay attention to the mental health of the children and their families and provide psychological counseling and support for them. The diagnosis of SMA often brings a heavy psychological burden to families. Psychologists help parents and children relieve negative emotions such as anxiety and fear through regular psychological counseling, and establish confidence in facing the disease positively.
Through long-term clinical practice, the hospital has formed a set of mature and effective SMA treatment plans. For diagnosed children with SMA, first, appropriate drug treatment plans are selected according to factors such as the condition and family economic status. When using Nusinersen Injection, the standardized injection procedures and doses are strictly followed to ensure that the drug can accurately act on the target and exert the best efficacy. At the same time, combined with active rehabilitation treatment, rehabilitation training is involved simultaneously from the time of the child's diagnosis, and the training plan is continuously adjusted according to the child's age, disease progression, and rehabilitation effect.
In terms of clinical effects, many children with SMA treated in Jingdu Children's Hospital have made encouraging progress. In terms of motor ability recovery, according to the hospital's clinical data statistics, among children with type I SMA who received standardized treatment, more than 60% had significantly improved head control ability after 6 months of treatment and could independently complete head-raising actions. For type II children who originally could only rely on wheelchairs, after about 1 year of treatment and rehabilitation training, nearly 50% had significantly enhanced upper limb strength, could independently perform simple daily activities such as grasping objects and using tableware to eat, and some children could even complete dressing actions with the help of assistive devices. Some children with milder type III SMA have more significant improvement in motor function under continuous treatment. Among children who originally could not stand or walk, about 30% could walk short distances with the help of assistive devices after 2 years of treatment, and a few children with better recovery could walk several meters independently without assistive devices.
In terms of survival rate and quality of life improvement, without effective treatment, most children with type I SMA cannot survive to the age of 2. However, the 5-year survival rate of type I children treated in Jingdu Children's Hospital has increased to more than 75%. These children not only have prolonged survival time but also significantly improved quality of life. The incidence of pulmonary infections caused by respiratory muscle weakness has decreased by 40%. The improvement of swallowing function has reduced choking during eating, and the problem of malnutrition has been effectively alleviated. Parents of the children also have high evaluations. Many parents said that after the child received treatment, not only did their physical condition improve, but their personality also became cheerful and lively. They could actively communicate and play with other children, and the family's living atmosphere has changed from the previous depression to full of hope.
Beijing Jingdu Children's Hospital is not satisfied with the existing treatment results but continues to explore and innovate in the field of SMA treatment. On the one hand, the hospital actively participates in domestic and foreign scientific research cooperation projects, carries out basic research and clinical trials on SMA with top medical institutions and research teams, and explores new treatment methods and drugs. For example, participating in the research of some underdeveloped gene therapy drugs, hoping to bring a more thorough cure plan for children with SMA. On the other hand, the hospital pays attention to summarizing clinical experience and shares its experience in SMA treatment with other medical institutions by holding academic conferences and training courses. The hospital's expert team is often invited to give speeches at domestic and foreign medical conferences, introducing successful cases and innovative practices of Jingdu Children's Hospital in SMA treatment, and promoting the development of the entire SMA treatment field.
In the treatment of childhood spinal muscular atrophy (SMA), Beijing Jingdu Children's Hospital, relying on the introduction of cutting-edge drugs, professional multidisciplinary teams, mature treatment plans, and continuous exploration and innovation spirit, has brought hope and light to many children with SMA and their families, becoming a leader in the field of SMA treatment in China, and its experience has also provided valuable reference for other hospitals.
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