| Publication |
Sentence |
Publish Date |
Extraction Date |
Species |
| Isabel Conceição, John L Berk, Markus Weiler, Pedro A Kowacs, Noel R Dasgupta, Sami Khella, Chi-Chao Chao, Shahram Attarian, T Jesse Kwoh, Shiangtung W Jung, Jersey Chen, Nicholas J Viney, Rosie Z Yu, Morie Gertz, Ahmad Masri, Márcia Waddington Cruz, Teresa Coelh. Switching from inotersen to eplontersen in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: analysis from NEURO-TTRansform. Journal of neurology. 2024-08-14. PMID:39138650. |
the phase 3 neuro-ttransform trial showed eplontersen treatment for 65 weeks reduced transthyretin (ttr), halted progression of neuropathy impairment, and improved quality of life (qol) in adult patients with hereditary ttr-mediated amyloidosis with polyneuropathy (attrv-pn), vs. historical placebo. |
2024-08-14 |
2024-08-16 |
Not clear |
| Chi-Chao Chao, Shiou-Ru Tzeng, Ming-Chang Chiang, Hsueh-Wen Hsueh, Wan-Jen Hsieh, Yuan-Chun Chao, Mei-Fang Cheng, Yen-Hung Lin, Mao-Yuan Su, Chun-Hsiang Huang, Yi-Shiang Wang, Ming-Fang Hsieh, Ping-Huei Tseng, Sung-Tsang Hsie. Diflunisal versus tafamidis on neuropathy and cardiomyopathy in hereditary transthyretin amyloidosis. Annals of clinical and translational neurology. 2024-08-03. PMID:39096004. |
hereditary transthyretin (ttr) amyloidosis (attrv) is frequently complicated by polyneuropathy (attrv-pn) and cardiomyopathy (attrv-cm). |
2024-08-03 |
2024-08-06 |
Not clear |
| Tsai-Jou Su, Chien-Ho Janice Lin, Yen-Lin Liu, Hsueh-Wen Hsueh, Sung-Tsang Hsieh, Chi-Chao Chao, Ming-Chang Chian. Altered connectivity of central autonomic network: effects of dysautonomia in hereditary transthyretin amyloidosis with polyneuropathy. Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis. 2024-07-24. PMID:39044725. |
hereditary transthyretin amyloidosis with polyneuropathy (attrv-pn) is a progressive fatal disorder caused by deposition of mutant transthyretin (ttr) amyloids mainly in the nerves and heart. |
2024-07-24 |
2024-07-26 |
Not clear |
| Gbolahan Olatunji, Emmanuel Kokori, Israel Charles Abraham, Oluwatobi Omoworare, Doyin Olatunji, Chimezirim Ezeano, Babawale Emmanuel Adeoba, Anthony Chidera Stanley, Awoyinfa Michael Oluwatobiloba, Omidiran Basit Oluwademilade, Kale Mekoya Shimelis, Olawale Olanisa, Nicholas Aderint. A mini-review of Vutrisiran and Eplontersen in hereditary transthyretin-mediated amyloidosis with polyneuropathy. Medicine. vol 103. issue 26. 2024-06-28. PMID:38941378. |
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. |
2024-06-28 |
2024-07-01 |
Not clear |
| Per M Roos, Sebastian K T S Wärmlände. Hereditary Transthyretin Amyloidosis (hATTR) with Polyneuropathy Clusters Are Located in Ancient Mining Districts: A Possible Geochemical Origin of the Disease. Biomolecules. vol 14. issue 6. 2024-06-27. PMID:38927056. |
hereditary transthyretin amyloidosis (hattr) with polyneuropathy (formerly known as familial amyloid polyneuropathy (fap)) is an endemic amyloidosis involving the harmful aggregation of proteins, most commonly transthyretin (ttr) but sometimes also apolipoprotein a-1 or gelsolin. |
2024-06-27 |
2024-06-29 |
Not clear |
| Silvia Vilches, María Martínez-Avial, Irene Méndez, Cristina Gómez González, María Ángeles Espinos. Sex Differences in Transthyretin Cardiac Amyloidosis: Unraveling the Complexities in Epidemiology, Pathophysiology, Diagnosis, and Treatment. Current heart failure reports. 2024-05-22. PMID:38775878. |
female sex is a predictor of a good response to tafamidis 20 mg in ttr polyneuropathy but otherwise there are no data on sex differences in the efficacy of other treatments for attr-ca. |
2024-05-22 |
2024-05-27 |
Not clear |
| Eugenia Conti, Sebastián Menazzi, Ana Mariel Finkelsteyn, María de Lourdes Figuerol. Late-onset familial amyloidosis polyneuropathy associated with c.186G>C in transthyretin. Revista de la Facultad de Ciencias Medicas (Cordoba, Argentina). vol 81. issue 1. 2024-03-27. PMID:38537102. |
familial amyloidosis polyneuropathy associated with variants of ttr (fap-ttr) is an infrequent, multisystemic disease, with predominant involvement of the peripheral nervous system. |
2024-03-27 |
2024-03-30 |
Not clear |
| Marco Luigetti, Dianna Quan, John L Berk, Isabel Conceição, Yohei Misumi, Chi-Chao Chao, Shaun Bender, Emre Aldinc, John Vest, David Adam. Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study. Neurology and therapy. 2024-03-21. PMID:38512694. |
vutrisiran, an rna interference therapeutic that results in rapid ttr knockdown, improved neuropathy and quality of life (qol) versus external placebo in patients with attrv amyloidosis with polyneuropathy in the phase 3 helios-a study (nct03759379). |
2024-03-21 |
2024-03-24 |
Not clear |
| Liqun Liang, Yuqi Zhang, Yanyan Zhu, Juxia Bai, Yangyang Ni, Junfeng Wan, Haiyan Yue, Qingjie Zhao, Huiyu L. Structures and Dynamics of β-Rich Oligomers of ATTR (105-115) Assembly. ACS chemical neuroscience. 2024-03-14. PMID:38483181. |
misfolding and aggregation of ttr can lead to amyloid transthyretin amyloidosis (attr), which can cause many diseases (e.g., senile systemic amyloidosis, familial amyloid cardiomyopathy, and familial amyloid polyneuropathy). |
2024-03-14 |
2024-03-16 |
Not clear |
| Bárbara Junqueira, Carlos Mestr. Iatrogenic Amyloid Polyneuropathy Following Domino Liver Transplantation: A Case Report. Cureus. vol 16. issue 2. 2024-03-07. PMID:38449948. |
in portugal, it is mainly linked to transthyretin (ttr) mutation, and patients present with length-dependent sensory-motor polyneuropathy, often accompanied by autonomic dysfunction. |
2024-03-07 |
2024-03-09 |
Not clear |
| Tina Ni. Eplontersen: First Approval. Drugs. 2024-02-27. PMID:38413492. |
subcutaneous eplontersen reduced serum ttr levels, inhibited neuropathy progression and improved health-related quality of life in patients with polyneuropathy of hereditary attr (attrv-pn; v for variant) in a phase iii trial. |
2024-02-27 |
2024-03-01 |
Not clear |
| Riccardo Sarzani, Francesco Spannella, Chiara Di Pentima, Federico Giulietti, Matteo Landolfo, Massimiliano Allev. Molecular Therapies in Cardiovascular Diseases: Small Interfering RNA in Atherosclerosis, Heart Failure, and Hypertension. International journal of molecular sciences. vol 25. issue 1. 2024-01-11. PMID:38203499. |
patisiran, binding transthyretin (ttr) mrna, has demonstrated an ability to improve heart failure and polyneuropathy in patients with ttr amyloidosis, even in older patients with wild-type form. |
2024-01-11 |
2024-01-13 |
Not clear |
| Juan Melesio, Bernardo Bonilauri, Audrey Li, Paul D Pang, Ronglih Liao, Ronald M Witteles, Joseph C Wu, Karim Salla. Generation of two induced pluripotent stem cell lines from hereditary amyloidosis patients with polyneuropathy carrying heterozygous transthyretin (TTR) mutation. Stem cell research. vol 74. 2023-12-15. PMID:38100909. |
generation of two induced pluripotent stem cell lines from hereditary amyloidosis patients with polyneuropathy carrying heterozygous transthyretin (ttr) mutation. |
2023-12-15 |
2023-12-18 |
Not clear |
| Juan Melesio, Bernardo Bonilauri, Audrey Li, Paul D Pang, Ronglih Liao, Ronald M Witteles, Joseph C Wu, Karim Salla. Generation of two induced pluripotent stem cell lines from hereditary amyloidosis patients with polyneuropathy carrying heterozygous transthyretin (TTR) mutation. Stem cell research. vol 74. 2023-12-15. PMID:38100909. |
hereditary transthyretin amyloidosis with polyneuropathy (attr-pn) results from specific ttr gene mutations. |
2023-12-15 |
2023-12-18 |
Not clear |
| Mitsuharu Ueda, Yohei Misumi, Toshiya Nomura, Masayoshi Tasaki, Shiori Yamakawa, Konen Obayashi, Taro Yamashita, Yukio And. Disease-Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy. Annals of neurology. 2023-12-06. PMID:38053464. |
hereditary transthyretin (attrv) amyloidosis is a rare, fatal systemic disease, associated with polyneuropathy and cardiomyopathy, that is caused by mutant transthyretin (ttr). |
2023-12-06 |
2023-12-10 |
Not clear |
| Mitsuharu Ueda, Yohei Misumi, Toshiya Nomura, Masayoshi Tasaki, Shiori Yamakawa, Konen Obayashi, Taro Yamashita, Yukio And. Disease-Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy. Annals of neurology. 2023-12-06. PMID:38053464. |
in addition to liver transplantation, several groundbreaking disease-modifying drugs (dmds) such as tetrameric ttr stabilizers and ttr gene-silencing therapies have been developed for attrv amyloid polyneuropathy. |
2023-12-06 |
2023-12-10 |
Not clear |
| Stacy Dixon, Xuan Kang, Dianna Qua. Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis. Therapeutics and clinical risk management. vol 19. 2023-12-04. PMID:38047038. |
variant transthyretin amyloidosis (attrv) is an autosomal dominant inherited genetic disorder that affects 5000-10,000 people worldwide. it is caused by mutations in the transthyretin (ttr) gene and results in amyloid deposition in a variety of organs due to abnormal accumulation of ttr protein fibrils. although this is a multisystem disorder, the heart and peripheral nerves are the preferentially affected organs. over 150 ttr gene mutations have been associated with this disease and the clinical phenotype can vary significantly. severe forms of the disorder can be fatal. fortunately, the oligonucleotide-based therapy era has resulted in the development of several novel treatment options. patisiran is a small interfering rna (sirna) encapsulated in a lipid nanoparticle that targets both mutant and wild-type ttr and results in significant reductions of the ttr protein in the serum and in tissue deposits. patisiran has been approved for treatment of adults with polyneuropathy due to hereditary ttr-mediated amyloidosis in both the united states (us) and european union (eu). in this review, we will discuss the development of patisiran, the clinical trials that lead to treatment approval, and provide guideline parameters for use in clinical practice. . |
2023-12-04 |
2023-12-10 |
Not clear |
| David Adams, Yoshiki Sekijima, Isabel Conceição, Marcia Waddington-Cruz, Michael Polydefkis, Andoni Echaniz-Laguna, Mary M Reill. Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment. The Lancet. Neurology. vol 22. issue 11. 2023-10-20. PMID:37863593. |
hereditary transthyretin (ttr) amyloid polyneuropathy is an autosomal dominant life-threatening disorder. |
2023-10-20 |
2023-11-08 |
Not clear |
| Vincenzo Di Stefano, Antonino Lupica, Paolo Alonge, Antonia Pignolo, Sofia Maria Augello, Francesca Gentile, Andrea Gagliardo, Francesca Giglia, Daniele Brinch, Maria Cappello, Daniela Di Lisi, Giuseppina Novo, Eugenia Borgione, Carmela Scuderi, Filippo Brighin. Genetic screening for hereditary transthyretin amyloidosis with polyneuropathy in western Sicily: Two years of experience in a neurological clinic. European journal of neurology. 2023-09-19. PMID:37725003. |
hereditary transthyretin amyloidosis with polyneuropathy (attrv-pn) is caused by mutations in the ttr gene, leading to misfolded monomers that aggregate generating amyloid fibrils. |
2023-09-19 |
2023-10-07 |
Not clear |
| Adam Ioannou, Marianna Fontana, Julian D Gillmor. Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy. Heart international. vol 17. issue 1. 2023-07-17. PMID:37456349. |
ttr can misfold into pathogenic amyloid fibrils that deposit in the heart and nerves, causing a life-threatening transthyretin amyloidosis cardiomyopathy (attr-cm), and a progressive and debilitating polyneuropathy (attr-pn). |
2023-07-17 |
2023-08-14 |
Not clear |