The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
Alnylam Pharmaceuticals (ALNY) is the sponsoring company for this trial. BiotechSign currently grades this company F (27/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.