Longitudinal Transcriptomic Analysis Reveals Systemic Effects of Risdiplam in Adults with Spinal Muscular Atrophy
Background: Spinal Muscular Atrophy (SMA) is a neurodegenerative disease caused by reduced survival motor neuron (SMN) protein levels due to <i>SMN1</i> gene mutations. The natural history of SMA has dramatically changed since innovative therapies were approved; among them, Risdiplam (an oral molecu...
Збережено в:
| Автори: | , , , , , , |
|---|---|
| Формат: | Artigo |
| Мова: | Inglês |
| Опубліковано: |
MDPI AG
2026-06-01
|
| Серія: | Brain Sciences |
| Предмети: | |
| Онлайн доступ: | https://www.mdpi.com/2076-3425/16/6/643 |
| Теги: |
Немає тегів, Будьте першим, хто поставить тег для цього запису!
|