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

Patients with Gaucher disease (GD), a rare lysosomal storage disorder, have reduced health-related quality of life (HRQOL). A patient-reported outcome measure (PROM) for HRQOL developed for type 1 GD (GD1) is ...

Source: Orphanet Journal of Rare Diseases - January 5, 2024 Category: Internal Medicine Authors: Aya Narita, Yuta Koto, Shinichi Noto, Masafumi Okada, Midori Ono, Terumi Baba, Rieko Sagara and Norio Sakai Tags: Research Source Type: research