General treatment 1. Respiratory care: Lung disease is the most important complication. It is also the cause of death in type I and type II SMA. A small number of patients with type III SMA are also affected. Severe muscle atrophy causes these patients to be confined to bed for long periods of time or to get up occasionally with assistance. They are unable to cough forcefully to expel respiratory secretions, so patients are prone to periodic pulmonary infections, which in turn can exacerbate the degree of muscle atrophy (especially in the respiratory muscles), leading to atelectasis, pulmonary atrophy, and a tendency to develop pulmonary hypoventilation at night. These patients may require rapid external provision of respiratory support such as mechanical ventilation and, if necessary, tracheotomy to save the patient’s life, but the ethics of this damaging approach is currently being debated in France. These patients need prompt antibiotic treatment, including appropriate vaccinations to prevent lung infection, to slow the progression of the disease and to improve the patient’s quality of life. Nutritional support: Children with SMA can develop a variety of gastrointestinal problems. Gastroesophageal reflux, constipation, abdominal distention and delayed gastric emptying are examples. Reflux is a major contributor to death. In severe cases, it can lead to respiratory arrest. In mild cases, aspiration pneumonia can result. Patients should avoid high-fat foods. This is because high-fat food delays gastric emptying and increases the chances of reflux. Medications used to treat GERD include acid neutralizers and gastric secretion inhibitors. Gastric power medications are not recommended due to the specific nature of the disease, and SMA patients should pay attention to weight control, based on weight-to-height ratio. Maintain a lower than normal weight to height ratio. Reasonable control of diet to avoid obesity. 2, rehabilitation training: Meldrum et al. reported that regular physical exercise can help children with SMA to strengthen muscle and joint strength and increase skeletal muscle density. Grondard et al. also found that regular exercise played a positive role in the type II SMA model rats, and the survival of the rats was significantly prolonged. Bone marrow motor neuron death was also greatly reduced. It is advisable to allow the affected children to perform regular exercise, such as swimming, in their daily life. Appropriate exercise is essential for restoring self-esteem, social integration and health. It is important to integrate into society and maintain physical health. The limitation of limb movement due to muscle atrophy. Ultimately, this can lead to spinal deformities. Difficulties in walking and performing activities of daily living, often with pain and fractures. Appropriate medical interventions such as postural correction, pain control, and contracture control can prolong the survival of the child. The burden of life can be reduced. Drug therapy There is no drug that can cure SMA disease. Drugs targeting the SMN2 gene have been used in SMA patients. Drugs that increase transcription levels and stabilize the corresponding proteins are also recommended. The following drug types are currently available. (1) Histidine deacetylase inhibitors: These drugs activate the transcription of the SMN2 gene. When histidine is acetylated. The transcription factor readily approaches some genes, including the SMN2 gene, thereby enhancing normal transcription. Valproic acid, sodium butyrate and phenethyl butyrate belong to this class of drugs. In particular, valproic acid and phenethyl butyrate can effectively cross the blood-brain barrier to reach the central nervous system: in valproic acid-stimulated in vitro experiments, an increase in motor neuron survival protein could be observed on hippocampal slices and motor neurons of type l model mice on fibroblasts from type I patients. In the in vivo experiments . The results showed an increase in the level of motor neuron survival protein in the spinal cord of the experimental rats compared with the control group. Motor function improved, neuronal degenerative lesions were reduced, and neuronal distribution in neuromuscular junctions was increased. a non-randomized controlled clinical study in a small number of patients by Tsai et al. found a slight increase in muscle strength and voluntary mobility (the dose of valproic acid was given as valproic acid for epilepsy). swoboda et al. selected 42 patients between 2 and 31 years of age with type I, II, and III SMA patients given valproic acid (15-50 mg.kg-1.d-1), due to the large variation in the case sample. The results were not very clear. By Rak et al. neuronal cells from type I SMA diseased rats were cultured in vitro and stimulated with valproic acid in vitro. The expression of motor neuron survival proteins was found to be elevated, but the physiological excitability of the axon terminals was reduced. Other drugs applied as preclinical non-histidine deacetylation inhibitors that can increase the level of censored gene transcription are hydroxyurea and quinazoline analogs. (2) Drugs that stabilize and increase SMN proteins: These drugs include indolov (non-steroidal anti-inflammatory class) and some aminoglycoside antibiotics, such as butamycin and tobramycin shirts. These drugs can increase the translation of motor neuron survival proteins and thus increase the stability of motor neuron survival proteins. However, both of these drugs are difficult to cross the blood-brain barrier into the central nervous system. This limits the clinical application. The effect of SMN protein enhancement has been demonstrated in animal experiments such as Riessland. (3) Neurotrophic factor drugs: Wang Xu et al. used murine nerve growth factor to treat SMA patients before and after their own controlled clinical study to observe the clinical efficacy and safety of murine nerve growth factor in treating SMA. 8 children completed the safety assessment. Seven of the children completed all efficacy assessments. No adverse effects occurred in all subjects; clinical symptoms improved to some extent, and muscle strength improved by 0.5 grade: the results of EMG and the assessment scale for patients with low capacity showed a trend of improvement. Neurotrophic factor slows motor neuron death and axonal degeneration. It has been reported to be a good therapeutic candidate in several motor neuron diseases. Stem cell transplantation and gene therapy Stem cells are primitive cells with self-replicating and multi-directional differentiation potential. Under certain conditions, it can differentiate into multiple functional cells or tissues and organs. Stem cell therapy is the transplantation of healthy stem cells into patients to repair diseased cells or rebuild functionally normal cells and tissues. Let stem cells differentiate a large number of motor neuron stem cells. Attempts to treat SMA. motor neurons and improved survival in SMA model rats. It was concluded that stem cells have a positive effect on improving the SMA disease phenotype. Gene therapy can also be used to alter the SMN2 splicing pattern or control the translation process. In 2010, Passini et al. found a significant amount of SMN protein expression in the spinal cord by injecting AAV8-hSMN into the CNS of SMA model rats. In addition, the skeletal muscles of the rats became more robust, including increased thickening of the muscle fibers, and the neuromuscular junction (NMJ) structure became more refined. The survival of the diseased rats was extended to 50 d, which was significantly better than that of the control group at 15 d. The same results were obtained by Foust et al. in 2010 using adenovirus containing SMN vector (scAAV9.SMN) for early intravenous treatment of model disc rats. The ability of scAAV9 to cross the blood-brain barrier in non-human SMA models makes scAAV9 a further step forward for clinical application. Burghes and McGovecnl26J mentioned the role of oligonucleotide chains (ASO) in altering gene splicing patterns and thereby increasing SMN protein, not only in increasing SMN protein levels7 but also in clarifying the toxic substances produced. 4.Therapy dependent on increasing fl-SMN protein Some recent studies based on SMA murine models have shown that the progression of degenerative lesions in motor neurons of SMA patients may be related to the functional defects of NMJ.com Bowerman et al. used Y.27632, an inhibitor of ROCK (an actin metabolic regulator downstream of the action pathway), in SMA murine models and found that it could indeed prolong the duration of SMA. found that it did prolong the survival of SMA mice, however, by detecting the ratio of fl-SMN to smn△7 mRNAs revealed that the ROCK inhibitor was directly responsible for prolonging the survival of SMA mice, but not due to the increased fl-SMN protein. Further studies showed that Y-27632 improved maturation by inhibiting ROCK. In addition there was a promotive effect on muscle fiber growth. Improving muscle fiber growth without increasing fl-SMN protein is an effective way to prolong SMA in rats, and is a new therapy worthy of consideration for most clinical patients who have degenerative neuronal disease and are too late to use fl-SMN protein enhancement methods. Summary The treatment of SMA is currently being investigated to delay further disease progression mainly by improving residual muscle function. With clinical care and neuronal recovery approaches, many SMA patients can achieve normal survival, and as research progresses. the treatment of SMA will certainly have a promising future.