China News Service, Shanghai, August 7th (Reporter Chen Jing) August 7th this year is the fifth International Spinal Muscular Atrophy (SMA) Care Day.
The first guidebook in the field of rare diseases in China that focuses on patient education and long-term development - the "Admission Guidelines for SMA School-aged Patients" (hereinafter referred to as the "Admission Guidelines") was officially released on the 7th. Diagnosis and rehabilitation are carried out in four aspects, providing practical guidance for school-age patients with SMA.
Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder characterized by progressive muscle weakness, lagging limbs, and muscle wasting.
Due to limited motor function, most SMA patients are unable to live independently, let alone receive a normal education and return to society.
However, the intelligence and cognitive levels of SMA patients were no different from those of ordinary children.
Now, with the inclusion of SMA disease-modifying treatments in Medicare, patient access to treatment has greatly improved.
More and more school-aged patients have received better diagnosis and treatment, achieved better physical function, and are more looking forward to starting or continuing their study and life.
However, the study status of this group of patients is not optimistic.
Feng Jiamei, the founder of Meier SMA Care Center, said in an interview on the 7th: "The right to education is one of the basic rights that every child should have. We have also noticed that many SMA patients of the right age suffer from subjective and objective reasons. There is a lot of resistance when applying for admission, and even have to drop out of school.” It is reported that Meier SMA Care Center is a patient organization focusing on SMA in China, and is committed to providing support and assistance for the SMA population throughout the life cycle. , which not only includes treatment and rehabilitation, but also includes the social integration of patients and the realization of personal values.
The "Admission Guide" is aimed at the actual difficulties in admission of SMA patients due to physical disabilities and other reasons, and invites industry experts to start from policy and legal basis, admission application, psychological construction, material preparation, diagnosis and treatment and rehabilitation, etc., and calls on schools and society for special children. Provide reasonable facilities to help SMA school-age patients and their parents to solve the actual enrollment problems, participate in and experience the process of socialization, so as to realize their life goals and self-worth.
Feng Jiamei believes that the launch of the "Admission Guide for SMA School-aged Patients" hopes to provide practical guidance and support for more school-aged SMA patients and their families, help them avoid detours, quickly and safely integrate into the classroom, master learning and social skills, enjoy Happy school.
Fan Xiaohong, chairman of Beijing Baixing Yizhong Legal Services and Research Center for the Disabled and a supervisor of the China Association for the Development of Disabled Persons, said: "The admission of SMA school-age patients is protected by relevant national laws and regulations. The location or the legal guardian's actual work and residence are nearest to the school."
The release of the "Admission Guidelines" not only provides practical guidance for patients who are enrolled in school or who drop out of school due to illness to return to school, but also provides more practical explorations for patients with rare diseases to gradually integrate into society.
The "Admission Guide" also makes specific suggestions on the convenience conditions that SMA patients can obtain for admission, such as consultation for patients, renovation of barrier-free facilities in schools, and special policies for high school and college entrance examinations.
Many experts believe that although learning knowledge is the main purpose of school-age patients with SMA, a good body is the foundation of all learning, and how to balance the scale between learning and rehabilitation is still a topic that patients must face.
Qin Lun, the head of rehabilitation medicine department of Peking University First Hospital, suggested: "Parents should make adequate material preparation for their children's travel, study, life and rest before entering school. In actual use, they mainly include wheelchairs and cushions. , suitable desk stationery and daily necessities, etc. In addition, for school-age patients, a good sitting posture is the key to active learning and prevention of scoliosis." In class sitting posture management and inter-class activities, some professional and practical action guidance has been sorted out, and supplemented by visual picture descriptions, which is convenient for patients and parents to refer to and learn.
Since SMA is a disease with progressive decline in motor function, considering the actual situation, school-age patients with SMA can only achieve long-term and regular learning on the premise of maintaining physical health and existing motor function.
Therefore, focusing on the treatment of school-age children and adolescents is also a key chapter in this "Admission Guide".
Mao Shanshan, an expert from the Children's Hospital Affiliated to Zhejiang University School of Medicine, and Jiang Haishan, an expert from the Southern Hospital of Southern Medical University, serve as the expert editorial board members of this chapter. professional advice.
They believe that for school-aged children, starting disease-modifying therapy as early as possible can help improve or preserve more motor function.
Mao Shanshan, chief physician of the Children's Hospital Affiliated to Zhejiang University School of Medicine, emphasized: "Because of the particularity of the disease, children with SMA need to regularly assess their physical conditions and receive treatment before and after they enter school, and at the same time formulate and adjust corresponding study plans. Therefore, we encourage children to start disease-modifying therapy as early as possible, follow up regularly and carry out multidisciplinary comprehensive management, and balance their studies well, which will be a constant topic."
It is understood that for school-aged adolescents and even adult patients, real-world evidence also shows that after receiving intrathecal injection, patients can significantly improve and stabilize motor function, lung function, bite force, etc.
"Patients receiving Nosinagen sodium treatment need to go to the hospital for injections regularly, which is also conducive to the development of multidisciplinary comprehensive management." Jiang Haishan, deputy chief physician of the Southern Hospital of Southern Medical University, said, "The maintenance treatment period is 3 times a year. It is convenient for school-aged patients to arrange treatment and study time reasonably. From the doctor's point of view, we hope to see more patients enter the campus from the family, approach the collective, and realize the transformation from 'natural person' to 'social person'. Because the ultimate goal of treatment , not only to treat the disease, but also to maximize the integration of patients into society.”
Wen Haoji, president of the Asia-Pacific region of a multinational pharmaceutical company participating in the "Admission Guide" project, said in an interview: "I hope more SMA patients and families can benefit from this guide. The company will also continue to work with all parties to actively explore rare The whole life cycle management model of "screening-diagnosing-treatment-protection-long-term development" of patients with the disease allows these "small groups" to truly integrate into our larger society." (End)