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Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives
Highly efficacious, potentially curative gene therapies holds immense clinical promise, but also present complex challenges. At the time of regulatory approval and health technology assessment (HTA), evidence of efficacy and safety of gene therapies is often uncertain. In addition, research, develop...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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IOS Press
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9697054/ https://www.ncbi.nlm.nih.gov/pubmed/36314216 http://dx.doi.org/10.3233/JND-221540 |