Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence
Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato, Dinko Vitezić, Slobodan Janković, Andrej Belančić, On Behalf Of The EACPT Early Career Clinical Pharmacologists Working Group · 2025
Read Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence by Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato, Dinko Vitezić, Slobodan Janković, Andrej Belančić, On Behalf Of The EACPT Early Career Clinical Pharmacologists Working Group (2025) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-12657850.txt.
Description
Spinal muscular atrophy is a severe neuromuscular disorder caused by mutations in the survival motor neuron 1 gene, leading to progressive motor neuron degeneration. Over the past decade, disease-modifying therapies targeting the survival motor neuron pathway—nusinersen, onasemnogene abeparvovec, and risdiplam—have significantly transformed the clinical landscape of spinal muscular atrophy.
Catalog record
| CREATOR | Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato, Dinko Vitezić, Slobodan Janković, Andrej Belančić, On Behalf Of The EACPT Early Career Clinical Pharmacologists Working Group |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 57 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |