Nosey sodium injection is an antisense oligonucleotide (ASO), which was developed by the proprietary antisense technology of ion is Company. It is designed to treat SMA caused by SMN protein deficiency caused by mutation or deletion of SMN 1 gene (located on chromosome 5q). Noxinasone sodium injection can change the splicing of SMN2 pre-mRNA, thus increasing the production of full-length SMN protein.
ASO is a synthetic short nucleotide chain, which can be used to selectively bind to target RNA and regulate gene expression. Through the use of this technology, it has been proved that Nosey sodium injection can increase the number of full-length SMN protein in SMA patients.
Noxii sodium injection is administered by intrathecal injection, and the drug is directly delivered to cerebrospinal fluid (CSF) around the spinal cord, which is the part where SMA users suffer from motor neuron degeneration due to insufficient SMN protein level.
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SMA is a rare hereditary neuromuscular disease, and the incidence of newborns is about 1.67%, which is characterized by symmetrical muscle weakness, muscle relaxation, muscle atrophy and severe limitation of limb motor function.
Hua Shaodong, chief physician of the neonatal intensive care unit of Bayi Children's Hospital affiliated to the Seventh Medical Center of PLA General Hospital, explained that it is difficult for users of spinal muscular atrophy to turn over, kick and crawl. With the development of the disease, it will eventually affect swallowing and breathing, threatening the life of users.
The diagnosis of this disease is easy in China, but there is a lack of effective treatment drugs. Before the introduction of Nosey sodium injection, SMA disease management was limited to respiratory support, nutritional support, prevention and treatment of serious complications caused by myasthenia gravis.
People's Daily Online-The Pediatric Department of Peking University First Hospital completed the first batch of intrathecal injection of Nocichine.