{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T20:10:32Z","timestamp":1792440632544,"version":"4.5.0"},"reference-count":27,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2026,2,18]],"date-time":"2026-02-18T00:00:00Z","timestamp":1771372800000},"content-version":"vor","delay-in-days":17,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2026,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Hereditary transthyretin (ATTRv) amyloidosis is a rare, genetically heterogeneous disease leading to an unstable configuration of transthyretin (TTR) and hence irregular deposition of TTR amyloid fibrils. The disease predominantly affects the peripheral nervous system, resulting in a progressive sensorimotor polyneuropathy (ATTRv\u2010PN), but can also involve the cardiac and other organ systems. The\n                      <jats:italic>TTR<\/jats:italic>\n                      mRNA silencers patisiran and inotersen effectively reduce serum TTR levels, thereby improving neurologic disability and quality of life in patients with ATTRv\u2010PN. This real\u2010world study investigated long\u2010term efficacy and tolerability of both compounds in patients with ATTRv\u2010PN.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This retrospective study from a German single\u2010referral center assessed the effects of treatment with patisiran or inotersen on clinical parameters of 35 patients with ATTRv\u2010PN treated at the Amyloidosis Center of Heidelberg University Hospital for up to 6\u2009years after therapy start. The study included analyses of serum TTR levels, NT\u2010proBNP, creatinine and modified body mass index (mBMI), and reported side effects throughout the observational period.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Both silencers stably reduced serum TTR levels by 81.1% (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.0039, patisiran) and 82.8% (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.0039, inotersen), respectively, after 8 to 15\u2009months compared to mean baseline. NT\u2010proBNP, creatinine, and mBMI values remained stable during treatment. With patisiran, a lower rate of reported adverse effects was observed. Grade 1 thrombocytopenia was reported primarily for inotersen\u2010treated patients.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>\n                      Our findings confirm long\u2010term treatment with\n                      <jats:italic>TTR<\/jats:italic>\n                      silencers in a real\u2010world setting is safe, consistently reducing serum TTR levels, stabilizing neurologic symptoms, cardiac and renal functions, and nutritional parameters.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.70529","type":"journal-article","created":{"date-parts":[[2026,2,18]],"date-time":"2026-02-18T09:15:45Z","timestamp":1771406145000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["First\u2010Generation\n                    <scp>\n                      <i>TTR<\/i>\n                    <\/scp>\n                    Silencing Therapies in Hereditary Transthyretin Amyloidosis With Polyneuropathy: Real\u2010World Insights From a German Single\u2010Referral Center"],"prefix":"10.1111","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8335-3543","authenticated-orcid":false,"given":"Marilin S.","family":"Koch","sequence":"first","affiliation":[{"name":"Amyloidosis Center Heidelberg University Hospital, and Faculty of Medicine, Heidelberg University  Heidelberg Germany"},{"name":"Department of Neurology Heidelberg University Hospital, and Faculty of Medicine, Heidelberg University  Heidelberg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4264-1722","authenticated-orcid":false,"given":"Fabian aus dem","family":"Siepen","sequence":"additional","affiliation":[{"name":"Amyloidosis Center Heidelberg University Hospital, and Faculty of Medicine, Heidelberg University  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