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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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Detalhes bibliográficos
Publicado no:Ther Innov Regul Sci
Autor principal: White, Molly
Formato: Artigo
Idioma:Inglês
Publicado em: Springer International Publishing 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7362889/
https://ncbi.nlm.nih.gov/pubmed/32557301
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s43441-019-00017-1
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