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Learnings from Patient-Report Workshop on Disease Progression in Myotonic Dystrophy
For researchers, pharmaceutical companies, and regulatory agencies involved in the development of treatments that slow or stop disease progression, the highly variable and unpredictable progression of symptoms in myotonic dystrophy (DM) makes it difficult to assess whether an intervention provides c...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2019
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7362889/ https://www.ncbi.nlm.nih.gov/pubmed/32557301 http://dx.doi.org/10.1007/s43441-019-00017-1 |