2020, Vol. 2, Issue 1, Part A

Our aim in this review is to discuss current treatments and investigational products for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and to provide suggestions for monitoring disease progression and treatment efficacy. We will explore the effectiveness of existing therapies, including pharmacological interventions and gene-silencing treatments, as well as emerging therapies currently under investigation. Additionally, we will address best practices for monitoring patients, including the use of biomarkers, imaging techniques, and clinical assessments to track disease progression and evaluate treatment responses. By synthesizing the latest research and clinical guidelines, we hope to offer a comprehensive resource for healthcare professionals managing ATTRv-PN.