Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials.

Deborah ElsteinNadia BelmatougPatrick DeeganÖzlem Göker-AlpanDerralynn A HughesIda Vanessa D SchwartzNeal WeinrebNicola BonnerCharlotte PanterDonna FountainAndrew LennyLouise LongworthRachael MillerKoonal ShahJörn SchenkRohini SenAri Zimran

Published in: Orphanet journal of rare diseases (2022)

Development of the ctGD1-PROM represents an important step forward for researchers measuring the impact of GD and its respective treatment.

Keyphrases

patient reported
clinical trial
patient reported outcomes
replacement therapy
study protocol