Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplan...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-12-01
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| Series: | Brain Sciences |
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| Online Access: | https://www.mdpi.com/2076-3425/10/12/952 |
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| author | Massimo Russo Luca Gentile Antonio Toscano M’Hammed Aguennouz Giuseppe Vita Anna Mazzeo |
| author_facet | Massimo Russo Luca Gentile Antonio Toscano M’Hammed Aguennouz Giuseppe Vita Anna Mazzeo |
| author_sort | Massimo Russo |
| collection | DOAJ |
| description | Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplantation (LT) and combined heart–liver transplantation, introduced in the 1990s, have been the only therapies for almost two decades. In 2011, tafamidis meglumine became the first specific drug approved by regulatory agencies, since then the attention toward this disease has progressively increased and several drugs with different mechanisms of action are now available. This review describes the drugs already on the market, those that have shown interesting results although not yet approved, and those currently being tested. |
| first_indexed | 2024-03-10T14:14:06Z |
| format | Article |
| id | doaj.art-eb1ecf720024411b8765ac80c6a8b566 |
| institution | Directory Open Access Journal |
| issn | 2076-3425 |
| language | English |
| last_indexed | 2024-03-10T14:14:06Z |
| publishDate | 2020-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Brain Sciences |
| spelling | doaj.art-eb1ecf720024411b8765ac80c6a8b5662023-11-20T23:58:11ZengMDPI AGBrain Sciences2076-34252020-12-01101295210.3390/brainsci10120952Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future ProspectsMassimo Russo0Luca Gentile1Antonio Toscano2M’Hammed Aguennouz3Giuseppe Vita4Anna Mazzeo5Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyUnit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, 98125 Messina, ItalyHereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplantation (LT) and combined heart–liver transplantation, introduced in the 1990s, have been the only therapies for almost two decades. In 2011, tafamidis meglumine became the first specific drug approved by regulatory agencies, since then the attention toward this disease has progressively increased and several drugs with different mechanisms of action are now available. This review describes the drugs already on the market, those that have shown interesting results although not yet approved, and those currently being tested.https://www.mdpi.com/2076-3425/10/12/952ATTRvamyloidosishereditary polyneuropathyTTR stabilizerTTR silencersgene therapy |
| spellingShingle | Massimo Russo Luca Gentile Antonio Toscano M’Hammed Aguennouz Giuseppe Vita Anna Mazzeo Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects Brain Sciences ATTRv amyloidosis hereditary polyneuropathy TTR stabilizer TTR silencers gene therapy |
| title | Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects |
| title_full | Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects |
| title_fullStr | Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects |
| title_full_unstemmed | Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects |
| title_short | Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects |
| title_sort | advances in treatment of attrv amyloidosis state of the art and future prospects |
| topic | ATTRv amyloidosis hereditary polyneuropathy TTR stabilizer TTR silencers gene therapy |
| url | https://www.mdpi.com/2076-3425/10/12/952 |
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