A mini-review of Vutrisiran and Eplontersen in hereditary transthyretin-mediated amyloidosis with polyneuropathy

被引:0
|
作者
Olatunji, Gbolahan [1 ]
Kokori, Emmanuel [1 ]
Abraham, Israel Charles [1 ]
Omoworare, Oluwatobi [2 ]
Olatunji, Doyin [3 ]
Ezeano, Chimezirim [4 ]
Emmanuel Adeoba, Babawale [5 ]
Stanley, Anthony Chidera [6 ]
Oluwatobiloba, Awoyinfa Michael [7 ]
Oluwademilade, Omidiran Basit [8 ]
Shimelis, Kale Mekoya [9 ]
Olanisa, Olawale [10 ]
Aderinto, Nicholas [11 ]
机构
[1] Univ Ilorin, Dept Med & Surg, Ilorin, Nigeria
[2] Lagos State Univ, Dept Med & Surg, Coll Med, Lagos, Nigeria
[3] Western Illinois Univ, Dept Hlth Sci, Macomb, IL USA
[4] Univ North Texas, Hlth Sci Ctr, Dept Hlth, Ft Worth, TX USA
[5] Griffith Coll Dublin, Dept Med & Surg, Dublin, Ireland
[6] Univ Calabar, Dept Med & Surg, Calabar, Nigeria
[7] Univ Lagos, Coll Med, Lagos, Nigeria
[8] Afe Babalola Univ, Dept Med Biochem, Ado Ekiti, Nigeria
[9] Addis Ababa Univ, Dept Med, Addis Ababa, Ethiopia
[10] Trinity Hlth Ctr, Dept Internal Med, Grand Rapids, MI USA
[11] Ladoke Akintola Univ Technol, Dept Med & Surg, Ogbomosho 210214, Nigeria
关键词
ATTRv amyloidosis polyneuropathy; Eplontersen; hereditary transthyretin-mediated amyloidosis; Vutrisiran;
D O I
10.1097/MD.0000000000038767
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis), known as Corino de Andrade disease, is a rare neurodegenerative disorder with a significant global impact characterized by the misfolding of transthyretin (TTR) protein leading to amyloid aggregation, ATTRv amyloidosis, especially with polyneuropathy, poses a considerable challenge in managing its rapid progression and debilitating effects. This mini-review focuses on the recent advancements in the treatment landscape for ATTRv amyloidosis with polyneuropathy, specifically the RNA interference therapeutic Vutrisiran and the ligand-conjugated antisense oligonucleotide Eplontersen. We aim to provide a comprehensive overview of the mechanisms, current evidence from clinical trials, and future directions for these novel therapeutic agents. Vutrisiran and Eplontersen have demonstrated significant clinical efficacy in improving neuropathic impairment, quality of life, and serum TTR levels in various trials. The distinct mechanistic approaches of these therapies, coupled with their acceptable safety profiles, offer promising avenues for addressing the complexities of ATTRv amyloidosis with polyneuropathy. The introduction of Vutrisiran and Eplontersen marks a pivotal moment in the quest for effective therapies against ATTRv amyloidosis with polyneuropathy. While clinical evidence is promising, ongoing research is crucial to deepen mechanistic understanding and address research gaps. Future perspectives include the potential expansion of therapeutic options and a more inclusive approach to cater to the diverse needs of individuals globally. This mini-review provides valuable insights into the evolving landscape of ATTRv amyloidosis management and sets the stage for further exploration in this challenging domain.
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页数:4
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