Spiranza (Nusinersen) 12mg/5mg injection
Minimum Order:: 1 box
Packaging Size:: Vial
Packaging Type:: Box
Spinraza® is the trade name of a medication that contains an active substance called nusinersen, a chemical belonging to the class of antisense nucleotides (ASO). ASO-s are sequences of nucleic acids mimicking human DNA.
Nusinersen has been designed to attach to the SMN2 gene and modify its structure. As a result, the SMN2 gene starts producing high amounts of the SMN protein. Abundance of SMN protein prevents further degeneration of motor neurons, which is a feature of SMA, and helps them regain lost function.
Spinraza has been proven unusually effective. In controlled clinical trials, more than 60% of treated children with the most severe form of SMA had the disease course reversed. This did not happen to a single child from the control group.
Treatment effects in SMA type 2 and weak 3 were even more astounding. When treated early, children who would barely be able to mainatin sitting position learned to stand and walk unsupported. Even ambulant teenage patients with SMA type 3 had their walking ability increased 3.5-fold after three years of treatment.
At the same time, those who have completely lost motor neurons or muscle tissue due to disease progression, especially in its most severe form, are not likely to see much benefit of this therapy. Like most drugs designed for SMA, Spinraza only improves the function of the existing motor neurons but does not grow new neurons or rebuild muscles.