Preclinical development of antisense conjugates for the treatment of myotonic dystrophy 1
<p>Myotonic dystrophy type 1 (DM1) is a complex autosomal dominant neuromuscular disorder that affects approximately 1 in 8,000 individuals. DM1 impacts multiple organ systems, including skeletal muscle, heart and central nervous system (CNS), leading to a diverse range of symptoms. Despite on...
| Autor principal: | |
|---|---|
| Altres autors: | |
| Format: | Thesis |
| Idioma: | English |
| Publicat: |
2024
|
| Matèries: |