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Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
• Despite the burden of gene therapy trials for DMD patients there is great enthusiasm. • Collaborating with relevant bodies (pharmacy) at an early stage can accelerate progress. • A hub and spoke model may be an option for delivering clinical trials and follow up care.
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| Publicado no: | Neuromuscul Disord |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Pergamon Press
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7564510/ https://ncbi.nlm.nih.gov/pubmed/33158687 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2020.10.001 |
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