Clinical ValidationSignificant clinical validation is still needed to better understand the efficacy and safety of PRX012.
Diagnosis ChallengesAmyloidosis remains a difficult to diagnose disease given that no single imaging, blood, or urine test is diagnostic for this disorder and that the presenting symptoms often mimic those of more common disorders.
PADOVA Study ResultsThe Phase 2b PADOVA study narrowly missed the statistical significance in the primary endpoint of time to confirmed motor progression.