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.