Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.

Karl Eugen MengelBruno BembiMireia Del ToroFederica DeodatoMatthias GautschiStephanie GrunewaldSabine GrønborgBénédicte HéronEsther M MaierAgathe RoubertieSaikat SantraAnna Tylki-SzymanskaSimon DayTara SymondsStacie HudgensMarc C PattersonChristina GuldbergLinda IngemannNikolaj H T PetersenThomas KirkegaardChristine Í Dali

Published in: Orphanet journal of rare diseases (2020)

CT-ORZY-NPC-001: ClincalTrials.gov NCT02435030, Registered 6 May 2015, https://clinicaltrials.gov/ct2/show/NCT02435030 ; EudraCT 2014-005,194-37, Registered 28 April 2015, https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-005194-37/DE . OR-REL-NPC-01: Unregistered.

Keyphrases

image quality
dual energy
computed tomography
contrast enhanced
positron emission tomography
study protocol
clinical trial
phase iii
magnetic resonance imaging
phase ii
open label