Oral risdiplam for specific therapy in adult patients with 5q spinal muscular atrophy in the Moscow region
| Autor: | Ekaterina S. Novikova |
|---|---|
| Jazyk: | English<br />Russian |
| Rok vydání: | 2023 |
| Předmět: | |
| Zdroj: | Анналы клинической и экспериментальной неврологии, Vol 17, Iss 3, Pp 88-93 (2023) |
| Druh dokumentu: | article |
| ISSN: | 2075-5473 2409-2533 |
| DOI: | 10.54101/ACEN.2023.3.11 |
| Popis: | 5q spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disease characterized by gradual loss of motor neurons with progressive muscle weakness and atrophy. A specific therapy has changed the prognosis for such patients, prevented worsening disability, and improved the quality of life. Here are presented follow-up data for 13 patients with SMA aged 1942 years receiving oral therapy for 20212023. Changes in motor functions were assessed using a Revised Upper Limb Module (RULM) every 6 months. According to the follow-up data for risdiplam use in adult patients with SMA in the Moscow region, condition can be stabilized and motor functions can be improved even in patients with a severe neurological deficit at advanced disease stages. |
| Databáze: | Directory of Open Access Journals |
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