Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan.

Aya NaritaYuta KotoShinichi NotoMasafumi OkadaMidori OnoTerumi BabaRieko SagaraNorio Sakai

Published in: Orphanet journal of rare diseases (2024)

We have developed a flexible and reliable PROM that can be tailored for use in all types of GD and propose using Parts 1 and 2 for GD1, Parts 2 and 3 for GD2, and Parts 1, 2, and 3 for GD3.

Keyphrases

patient reported outcomes
smoking cessation
physical activity
depressive symptoms
subarachnoid hemorrhage