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

Platt FM., Cortina Borja M., te Vruchte D., Mengel E., Amraoui Y., Imrie J., Jones S., Dali C., Fineran P., Kirkegaard T., Runz H., Lachman R., Bremova T., Strupp M.

Type

Journal article

Journal

Orphanet Journal of Rare Diseases

Publisher

BioMed Central

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