It is the only drug in the world approved for the treatment of SMA, an antisense oligonucleotide, which is applied intrathecal delivery method of treatment.SMA is deficient in SMN proteins due to mutations or deletions in the SMN1 gene, and Nocinazen sodium injection can alter the splicing of SMN2 pre-mRNA, thereby increasing the production of full-length SMN proteins.
In current clinical trials in more than 40 countries and territories, nocinafosine sodium injection has shown a favorable benefit-risk ratio, but it is also associated with common respiratory infections, as well as constipation as a side effect.
Method of administration
The drug is administered intrathecally via lumbar puncture, and treatment should be performed by a healthcare professional experienced in lumbar puncture.
Using a spinal puncture needle, the product is injected intrathecally for 1-3 minutes; it should not be injected in areas where skin infection or inflammation is present. Drainage of the same volume of cerebrospinal fluid (CSF) as the injected drug is recommended prior to administration.
Sedation may be required for administration of this product depending on the patient's clinical condition. The use of ultrasound (or other imaging techniques) to guide intrathecal administration may be considered, particularly in younger patients and in patients with scoliosis. Aseptic techniques should be used in the preparation and administration of this product.
The above reference? Baidu Encyclopedia-Nocinasone Sodium Injection