Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

This 2-part first in human (FIH) study consists of an open-label, single ascending dose Part 1, which may identify the optimal biologically active dose (OBD)

The NEURO-TTRansform Study

The NEURO-TTRansform Study is a Phase 3 clinical trial of an investigational antisense drug called AKCEA-TTR-LRx for people living with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). If you

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