{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T07:02:07Z","timestamp":1789542127471,"version":"build-2803163510"},"reference-count":41,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T00:00:00Z","timestamp":1789430400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T00:00:00Z","timestamp":1789430400000},"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":["Muscle and Nerve"],"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>\n                      Transthyretin (\n                      <jats:styled-content style=\"fixed-case\">TTR<\/jats:styled-content>\n                      ) stabilizers and gene\u2010silencing therapies are approved for hereditary transthyretin\u2010mediated amyloidosis with polyneuropathy (\n                      <jats:styled-content style=\"fixed-case\">ATTRv<\/jats:styled-content>\n                      \u2010\n                      <jats:styled-content style=\"fixed-case\">PN<\/jats:styled-content>\n                      ), but data from clinical practice in patients who transition between therapies due to disease progression remains limited. The primary aim of this study is to evaluate the effectiveness of patisiran in\n                      <jats:styled-content style=\"fixed-case\">ATTRv<\/jats:styled-content>\n                      \u2010\n                      <jats:styled-content style=\"fixed-case\">PN<\/jats:styled-content>\n                      patients who progressed despite prior treatment with tafamidis 20\u2009mg.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We conducted a retrospective multicenter observational study including patients with ATTRV30M (p.V50M) with polyneuropathy who were initially treated with tafamidis 20\u2009mg and subsequently switched to patisiran due to documented neuropathy progression. Patients had at least 12\u2009months of follow\u2010up under each treatment. Neuropathy progression was assessed using the Neuropathy Impairment Score (NIS), neurophysiological composite scores, nutritional status, serum biomarkers, and patient\u2010reported outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Thirty\u2010two patients (mean age 52\u2009\u00b1\u200914\u2009years; mean disease onset 43\u2009\u00b1\u200923.5\u2009years) were included. Median disease duration before treatment initiation was 1.8\u2009\u00b1\u20091.9\u2009years. The mean duration of tafamidis and patisiran treatment was 4.5\u2009\u00b1\u20092.8 and 2.5\u2009\u00b1\u20092.3\u2009years, respectively. The annual increase in NIS was significantly lower during patisiran treatment than during tafamidis (0.7\u2009\u00b1\u20093.9 vs. 3.9\u2009\u00b1\u20094.7;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.004). Decline in neurophysiological parameters slowed after switching.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>In this cohort, patisiran attenuated neuropathy progression in patients who had deteriorated despite tafamidis. These findings highlight the importance of ongoing clinical monitoring to guide timely treatment optimization.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70406","type":"journal-article","created":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T06:03:17Z","timestamp":1789538597000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Neuropathy Progression in Variant Transthyretin Amyloidosis: Comparison Between\n                    <scp>TTR<\/scp>\n                    Stabilizers and Gene Silencer Therapies"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6194-9787","authenticated-orcid":false,"given":"Catarina","family":"Falc\u00e3o de Campos","sequence":"first","affiliation":[{"name":"Department of Neurology Hospital Santa Maria  Lisbon Portugal"},{"name":"Centro de Estudos Egas Moniz, Faculdade de Medicina da Universidade de Lisboa  Lisbon Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5572-5242","authenticated-orcid":false,"given":"Isabel","family":"de Castro","sequence":"additional","affiliation":[{"name":"Department of Neurology Hospital Santa Maria  Lisbon Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8984-475X","authenticated-orcid":false,"given":"Jos\u00e9","family":"Castro","sequence":"additional","affiliation":[{"name":"Department of Neurology Hospital Santa Maria  Lisbon Portugal"},{"name":"Centro de Estudos Egas Moniz, Faculdade de Medicina da Universidade de Lisboa  Lisbon Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"In\u00eas Losada","family":"Lopez","sequence":"additional","affiliation":[{"name":"Servicio de Medicina Interna Hospital Universitario Son Llatzer, Instituto de Investigaci\u00f3n Sanitaria Illes Balears  Palma Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8046-112X","authenticated-orcid":false,"given":"Juan Gonzalez","family":"Moreno","sequence":"additional","affiliation":[{"name":"Servicio de Medicina Interna Hospital Universitario Son Llatzer, Instituto de Investigaci\u00f3n Sanitaria Illes Balears  Palma Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0934-9631","authenticated-orcid":false,"given":"Isabel","family":"Concei\u00e7\u00e3o","sequence":"additional","affiliation":[{"name":"Department of Neurology Hospital Santa Maria  Lisbon Portugal"},{"name":"Centro de Estudos Egas Moniz, Faculdade de Medicina da Universidade de Lisboa  Lisbon Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,9,15]]},"reference":[{"issue":"7","key":"e_1_2_14_2_1","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1038\/s41582-019-0210-4","article-title":"Hereditary Transthyretin Amyloidosis: A Model of Medical Progress for a Fatal Disease","volume":"15","author":"Adams D.","year":"2019","journal-title":"Nature Reviews. 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