Hereditary transthyretin-mediated amyloid polyneuropathy is a rare, inherited, and progressive disease affecting an estimated 10,000 people worldwide1
Hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) is the result of an inherited genetic mutation that causes a protein called transthyretin (TTR) to misfold into clusters called amyloid deposits.
These amyloid deposits build up in the body’s peripheral nerves, causing debilitating nerve damage throughout the body and resulting in progressive loss of neurological functions, such as loss of feeling and movement.
Amyloid deposits also accumulate in other major organs, compromising their function and causing extensive damage—this damage can be disabling and life-threatening.
What can you do?
The NEURO-TTRansform study is currently enrolling to evaluate the safety and effectiveness of an investigational antisense medication for people living with hATTR-PN.
- Coelho T, Ericzon BG, Falk R, et al. A Physician’s Guide to Transthyretin Amyloidosis. 2008.