Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD), two of the most common, child onset, rare neuromuscular disorders, present a case study for the translation of preclinical research into clinical work. Over the past decade, well-designed clinical trials and innovative methods have...

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Detalles Bibliográficos
Autores principales: Stimpson, Georgia, Chesshyre, Mary, Baranello, Giovanni, Muntoni, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9855753/
https://www.ncbi.nlm.nih.gov/pubmed/36687260
http://dx.doi.org/10.3389/fgene.2021.759994

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9855753/
https://www.ncbi.nlm.nih.gov/pubmed/36687260
http://dx.doi.org/10.3389/fgene.2021.759994

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