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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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Detalles Bibliográficos
Autor principal:	Landfeldt, Erik
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2022
Materias:	Review
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

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