Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management

Abstract

Valentina Vélez-Santamaría,1– 3 Velina Nedkova-Hristova,1 Moisés Morales de la Prida,1 Carlos Casasnovas1– 3 1Neuromuscular Unit, Neurology Department, Bellvitge University Hospital, Barcelona, Spain; 2Neurometabolic Diseases Laboratory, Bellvitge Biomedical Research Institute (IDIBELL), Barcelona, Spain; 3Centre for Biomedical Research on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Madrid, SpainCorrespondence: Valentina Vélez-Santamaría, Neurometabolic Diseases Laboratory, IDIBELL, Hospital Duran i Reynals, Gran Via 199, 08908 L’Hospitalet de Llobregat, Barcelona, Spain, Tel [phone], Fax [phone], Email [email protected]: 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: polyneuropathy, transthyretin, amyloid, transthyretin stabilizers, gene silencers, CRISPR/Cas9

Keywords

• [polyneuropathy
• transthyretin
• amyloid
• transthyretin stabilizers
• gene silencers
• crispr/cas9 ].