Rare Hereditary Clinical Trial Case Study

Running across 51 sites spanning the US, Europe, Brazil and Sweden the ongoing Phase 3, open-Label, randomized study aims to evaluate the efficacy and safety of an RNA targeted IMP in patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (hATTR-PN).

Through a combination of lab and home visits, over a period of 8+ years the research is reviewing changes from baseline of a range of values including TTR concentration, neuropathy impairment, overall disability scores, 10-meter walk tests and historical placebo controls.

Read the available case study to see how MRN's patient centered approach to decentralized trials and specialist site healthcare professionals is enabling the study to be rolled out globally to approximately 140 eligible male and female patients, aged between 18 and 82.