Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2
Spinal muscular atrophy (SMA) is a rare, autosomal recessive neuromuscular disorder and a leading genetic cause of infant mortality. The past decade has witnessed a paradigm shift in SMA management with the advent of disease-modifying drugs (DMDs). This narrative review aims to (i) summarize pivotal...
Sábháilte in:
| Príomhchruthaitheoirí: | , , , , , |
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| Formáid: | Artigo |
| Teanga: | Inglês |
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MDPI AG
2025-08-01
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| Sraith: | Biomedicines |
| Ábhair: | |
| Rochtain ar líne: | https://www.mdpi.com/2227-9059/13/8/1939 |
| Clibeanna: |
Níl clibeanna ann, Bí ar an gcéad duine le clib a chur leis an taifead seo!
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