Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management

Valentina Vélez-Santamaría; Velina Nedkova-Hristova; Moisés Morales de la Prida; Carlos Casasnovas

doi:10.2147/IJGM.S338430

Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management

Int J Gen Med. 2022 Dec 20:15:8677-8684. doi: 10.2147/IJGM.S338430. eCollection 2022.

Authors

Valentina Vélez-Santamaría^{1

2

3}, Velina Nedkova-Hristova¹, Moisés Morales de la Prida¹, Carlos Casasnovas^{1

2

3}

Affiliations

¹ Neuromuscular Unit, Neurology Department, Bellvitge University Hospital, Barcelona, Spain.
² Neurometabolic Diseases Laboratory, Bellvitge Biomedical Research Institute (IDIBELL), Barcelona, Spain.
³ Centre for Biomedical Research on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Madrid, Spain.

Abstract

Our aim in this review is to discuss current treatments and investigational products and their effect on patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and provide suggestions for monitoring disease progression and treatment efficacy.

Keywords: CRISPR/Cas9; amyloid; gene silencers; polyneuropathy; transthyretin; transthyretin stabilizers.

Publication types

Review