Gaucher Disease Clinical Trial Patient Recruitment

Gaucher disease patient recruitment for Type 1, 2, and 3 ERT, SRT, and gene therapy clinical trials.

Who qualifies: Patients with confirmed Gaucher disease (GBA mutation) across all types, treatment-naive and ERT-experienced.

Recruitment challenges: GBA mutation confirmation and glucocerebrosidase enzyme activity requirements.

Our approach: Metabolic disease center networks and Gaucher patient advocacy partnerships.