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Gene Silencing Therapy in Hereditary (Variant) Transthyretin Cardiac Amyloidosis: A Puzzling Case of Decreasing Pyrophosphate Uptake on Scintigraphy.

作者信息

Smiley Dia A, Einstein Andrew J, Mintz Akiva, Shetty Mrinali, Chan Nicholas, Helmke Stephen T, Bhatia Ketan, Goldner Kim, Brannagan Thomas H, Santana Denisse D, Maurer Mathew S

机构信息

Seymour, Paul, and Gloria Milstein Division of Cardiology, Department of Medicine (D.A.S., A.J.E., M.S., N.C., S.T.H., D.D.S., M.S.M.), Columbia University Irving Medical Center, New York-Presbyterian Hospital.

Department of Radiology (A.J.E., A.M., K.B., K.G.), Columbia University Irving Medical Center, New York-Presbyterian Hospital.

出版信息

Circ Cardiovasc Imaging. 2023 Aug;16(8):e015243. doi: 10.1161/CIRCIMAGING.123.015243. Epub 2023 Jun 19.

DOI:10.1161/CIRCIMAGING.123.015243
PMID:37334703
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10524886/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/93cb1b0596b3/nihms-1903440-f0003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/0eaff94241cb/nihms-1903440-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/8710ad264e4d/nihms-1903440-f0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/93cb1b0596b3/nihms-1903440-f0003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/0eaff94241cb/nihms-1903440-f0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/8710ad264e4d/nihms-1903440-f0002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6883/10524886/93cb1b0596b3/nihms-1903440-f0003.jpg

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Gene Silencing Therapy in Hereditary (Variant) Transthyretin Cardiac Amyloidosis: A Puzzling Case of Decreasing Pyrophosphate Uptake on Scintigraphy.遗传性（变异型）转甲状腺素蛋白心脏淀粉样变性的基因沉默治疗：一例骨闪烁显像中焦磷酸盐摄取减少的疑难病例
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引用本文的文献

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Impact of Tafamidis on [Tc]Tc-pyrophosphate Scintigraphy in Ala97Ser Hereditary Transthyretin amyloid cardiomyopathy: significant initial reduction with stable Long-Term effects.他法米地对Ala97Ser遗传性转甲状腺素蛋白淀粉样心肌病患者[锝]锝-焦磷酸盐闪烁扫描的影响：初期显著降低，长期效果稳定
Eur J Nucl Med Mol Imaging. 2025 Apr;52(5):1853-1863. doi: 10.1007/s00259-025-07079-4. Epub 2025 Jan 13.
2
Use of Technetium-99m-Pyrophosphate Single-Photon Emission Computed Tomography/Computed Tomography in Monitoring Therapeutic Changes of Eplontersen in Patients With Hereditary Transthyretin Amyloid Cardiomyopathy.应用锝-99m-焦磷酸盐单光子发射计算机断层扫描/计算机断层扫描监测遗传性转甲状腺素蛋白淀粉样心肌病患者依罗替尼治疗的变化。
J Am Heart Assoc. 2024 Jan 16;13(2):e030512. doi: 10.1161/JAHA.123.030512. Epub 2024 Jan 12.

本文引用的文献

1
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.用于治疗遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病患者的 vutrisiran 的疗效和安全性：一项随机临床试验。
Amyloid. 2023 Mar;30(1):1-9. doi: 10.1080/13506129.2022.2091985. Epub 2022 Jul 23.
2
Cardiac microcalcifications in transthyretin (ATTR) amyloidosis.心脏微钙化在转甲状腺素蛋白（ATTR）淀粉样变性。
Int J Cardiol. 2022 Apr 1;352:84-91. doi: 10.1016/j.ijcard.2022.01.036. Epub 2022 Jan 22.