Accelerating Rare Disease Drug Development: Lessons Learned from Muscular Dystrophy Patient Advocacy Groups

With scientific and molecular advancements related to disease pathogenesis, advances in gene and stem cell therapies, and the promise of lucrative markets for biopharmaceutical companies, there has been a rapid expansion in the number of potential new muscular dystrophy (MD) treatments. The first ch...

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Detalles Bibliográficos
Autores principales: Huml, Raymond A., Dawson, Jill, Bailey, Michelle, Nakas, Nermina, Williams, Jane, Kolochavina, Maryna, Huml, Jonathan R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2020
Materias:
Analytical Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7513900/
https://www.ncbi.nlm.nih.gov/pubmed/32974874
http://dx.doi.org/10.1007/s43441-020-00221-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7513900/
https://www.ncbi.nlm.nih.gov/pubmed/32974874
http://dx.doi.org/10.1007/s43441-020-00221-4

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