Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration

Substantial challenges in study design and methodology exist during clinical trial development to examine treatment response in patients with a rare disease, especially those with predominant central nervous system involvement and heterogeneity in clinical manifestations and natural history. Here we...

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Detalles Bibliográficos
Autores principales: Videnovic, Aleksandar, Pfeiffer, Helle C. V., Tylki-Szymańska, Anna, Berry-Kravis, Elizabeth, Ezgü, Fatih, Ganju, Jitendra, Jurecka, Agnieszka, Lang, Anthony E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10032345/
https://www.ncbi.nlm.nih.gov/pubmed/36970548
http://dx.doi.org/10.3389/fneur.2023.1098454

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10032345/
https://www.ncbi.nlm.nih.gov/pubmed/36970548
http://dx.doi.org/10.3389/fneur.2023.1098454

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