Drug with Active Substance Nusinersen 12 Mg, Solution for Injection Drug with Active Substance Nusinersen 12 Mg, Solution for Injection

DRUG WITH ACTIVE SUBSTANCE NUSINERSEN 12 MG, SOLUTION FOR INJECTION DRUG WITH ACTIVE SUBSTANCE NUSINERSEN 12 MG, SOLUTION FOR INJECTION
The subject of the order is the drug NUSINERSEN 12 MG INJECTION SOLUTION Supplement: salt inj 5 ml/12 mg (liek.inj.skl.) Administration: intrathecal use One injection vial with a volume of 5 ml contains sodium salt of nusinersen, which corresponds to 12 mg of nusinersen. One ml contains 2.4 mg of nusinersen. Nusinersen is an antisense oligonucleotide (ASO) that increases the proportion of exon 7 inclusion in messenger ribonucleic acid (mRNA) transcripts for SMN2 (survival motor neuron 2, SMN2) by binding to the intronic splice silencing site (ISS-N1) located in intron 7 of SMN2 pre-messenger ribonucleic acid (pre-mRNA) for SMN2. With this binding, ASO displaces splicing factors that normally suppress splicing. Displacement of these factors results in retention of exon 7 in SMN2 mRNA, and therefore when SMN2 mRNA is formed, it can be transcribed into functional full-length SMN protein. The drug with the active substance Nusinersen is indicated for the treatment of 5q spinal muscular atrophy. Treatment with this medicine should only be started by a doctor with experience in spinal muscular atrophy (SMA). The decision to treat must be based on an individualized professional assessment of the expected benefits of treatment for a specific patient, balanced against the potential risk of drug treatment. In patients with severe hypotonia and respiratory failure at birth, in whom this drug has not been studied, there may not be a clinically significant benefit due to a severe deficiency of the SMN protein. The recommended dose is 12 mg (5 ml) per administration. Treatment should be started as soon as possible after diagnosis with 4 loading doses on days 0, 14, 28 and 63. A maintenance dose should then be given every 4 months.