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Areas of improvement in the medical care of SMA: evidence from a nationwide patient registry in Germany
BACKGROUND: Management and treatment of spinal muscular atrophy (SMA) has changed in recent years due to the introduction of novel transformative and potentially curative therapies resulting in the emergence of new disease phenotypes. Yet, little is known about the uptake and impact of these therapi...
| Autores principales: | , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9945617/ https://www.ncbi.nlm.nih.gov/pubmed/36810103 http://dx.doi.org/10.1186/s13023-023-02639-z |