Outcome measures for children with mitochondrial disease: consensus recommendations for future studies from a Delphi-based international workshop

Although there are no effective disease-modifying therapies for mitochondrial diseases, an increasing number of trials are being conducted in this rare disease group. The use of sensitive and valid endpoints is essential to test the effectiveness of potential treatments. There is no consensus on whi...

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Detalles Bibliográficos
Autores principales: Koene, Saskia, van Bon, Lara, Bertini, Enrico, Jimenez-Moreno, Cecilia, van der Giessen, Lianne, de Groot, Imelda, McFarland, Robert, Parikh, Sumit, Rahman, Shamima, Wood, Michelle, Zeman, Jiri, Janssen, Anjo, Smeitink, Jan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6326961/
https://www.ncbi.nlm.nih.gov/pubmed/30027425
http://dx.doi.org/10.1007/s10545-018-0229-5

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