Publications by authors named "K A Dolotov"
[Experience of the treatment of spinal muscular atrophy type 3 Kugelberg-Welander with Nusinersen].
Zh Nevrol Psikhiatr Im S S Korsakova
April 2024
Article Synopsis
- Before pathogenetic therapy, a diagnosis of spinal muscular atrophy (SMA) meant a grim outlook with inevitable decline and early death for patients.
- New therapies like nusinersen and risdiplam, along with onasemnogene abeparvovec, are now available in Russia, offering hope for SMA patients.
- Nusinersen works by modifying the splicing of genes to boost the production of a crucial protein for motor neuron survival, and the article highlights a successful case of long-term treatment in a patient with SMA type 3.