Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

BACKGROUND: Niemann-Pick disease type C (NPC) is a lysosomal storage disease with a heterogeneous neurodegenerative clinical course. Multiple therapies are in clinical trials and inclusion criteria are currently mainly based on age and neurological signs, not taking into consideration differential i...

Descripción completa

Detalles Bibliográficos
Autores principales: Cortina-Borja, Mario, te Vruchte, Danielle, Mengel, Eugen, Amraoui, Yasmin, Imrie, Jackie, Jones, Simon A., i Dali, Christine, Fineran, Paul, Kirkegaard, Thomas, Runz, Heiko, Lachmann, Robin, Bremova-Ertl, Tatiana, Strupp, Michael, Platt, Frances M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6097294/
https://www.ncbi.nlm.nih.gov/pubmed/30115089
http://dx.doi.org/10.1186/s13023-018-0880-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6097294/
https://www.ncbi.nlm.nih.gov/pubmed/30115089
http://dx.doi.org/10.1186/s13023-018-0880-9

Ejemplares similares