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INN: nusinersen

Indications for use

Nusinersen is an antisense oligonucleotide indicated for the treatment of spinal muscular atrophy.

Spinal muscular atrophy is a progressive neuromuscular disease with an autosomal recessive pattern of inheritance. The development of the disease is caused by mutations in the SMN1 (survival motor neuron) gene, which encodes the survival motor neuron protein. A decrease in the level of this protein leads to axonal defects in motor neurons and disorders in neuromuscular synapses, which is manifested by the development of progressive muscle atrophy, flaccid paresis and the development of complications in the musculoskeletal, respiratory and digestive systems. Nusinersen is a pathogenetic therapy drug that increases the amount of motor neuron survival protein, which prevents the progression of the disease and helps stabilize and/or improve the motor function of patients. Nusinersen has demonstrated robust and convincing efficacy across a broad, high-quality clinical trial program and has a well-studied, favorable safety profile. Lantesens is the first generic drug of nusinersen both in the Russian Federation and in the world. As part of this project in Russia, the GENERIUM company for the first time carried out the chemical synthesis of an oligonucleotide.

LP-No.(005199)-(RG-RU) dated 04/15/2024

Spinal muscular atrophy (SMA) 5q in adults and children.