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Res. Pract."],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Hereditary transthyretin (ATTRv) amyloidosis is a rare, progressive multisystem disease caused by pathogenic variants in the\n                    <jats:italic>transthyretin<\/jats:italic>\n                    (\n                    <jats:italic>TTR<\/jats:italic>\n                    ) gene. ATTRv-associated Polyneuropathy (ATTRv-PN) manifests in a length-dependent sensorimotor and autonomic pattern, commonly accompanied by cardiomyopathy. In recent years, clinical trials have introduced two classes of disease modifying treatments (DMT), TTR stabilizers and gene silencers. Across populations and outcome parameters, all trials share one\u00a0result\u00a0disease progression was significantly slowed and patients\u2019 span and quality of life substantially improved compared to placebo, even though a function once lost would not be retrieved. This expert consensus paper provides recommendations for the treatment of ATTRv-PN in Germany and was jointly developed by experts from the German Society of Amyloid Diseases (DGAK) and the German Neurological Society (DGN). In addition to evidence from pivotal clinical trials, the recommendations incorporate benefit assessments of the German Federal Joint Committee (G-BA). For first-line treatment, the DGAK\/DGN recommends one of the two second-generation\n                    <jats:italic>TTR<\/jats:italic>\n                    gene-silencing agents, vutrisiran or eplontersen, for most patients with symptomatic ATTRv-PN. For patients showing even minor signs of disease progression under a TTR stabilizer, the authors recommend immediate switch to a gene silencer. Evidence is limited for or against switching between silencers or combining different agents. Informed patient participation is crucial upon initiation and adaptation of DMT. For central nervous system (CNS) involvement, none of the approved agents sufficiently crosses the blood-brain barrier. Aligned with that, there are no observed or expected CNS side effects of any of the DMTs. In the future, next-generation RNA-interference therapies, gene-editing approaches, and amyloid-depleting antibodies are expected to further expand therapeutic options.\n                  <\/jats:p>","DOI":"10.1186\/s42466-026-00521-4","type":"journal-article","created":{"date-parts":[[2026,8,11]],"date-time":"2026-08-11T12:23:41Z","timestamp":1786451021000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Disease modifying treatment of hereditary transthyretin amyloidosis with polyneuropathy in Germany \u2013 expert consensus of the German society of amyloid diseases (DGAK) and the German neurological society (DGN)"],"prefix":"10.1186","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8942-7662","authenticated-orcid":false,"given":"Markus","family":"Weiler","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5320-9807","authenticated-orcid":false,"given":"Maike F.","family":"Dohrn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6055-5695","authenticated-orcid":false,"given":"Stefan","family":"Gingele","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8550-335X","authenticated-orcid":false,"given":"Thomas","family":"Skripuletz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-6806-3382","authenticated-orcid":false,"given":"Matthias","family":"Schilling","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3631-3450","authenticated-orcid":false,"given":"Tim","family":"Hagenacker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6158-1190","authenticated-orcid":false,"given":"Ernst","family":"Hund","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5787-6562","authenticated-orcid":false,"given":"Juliane","family":"Sachau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9492-4698","authenticated-orcid":false,"given":"Ralf","family":"Baron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-8711-5526","authenticated-orcid":false,"given":"Helena","family":"Pernice","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0987-8007","authenticated-orcid":false,"given":"Sebastian","family":"Spethmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4655-6269","authenticated-orcid":false,"given":"Cyrus","family":"Khandanpour","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3316-9166","authenticated-orcid":false,"given":"Alexander","family":"Carpinteiro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0920-6094","authenticated-orcid":false,"given":"Caroline","family":"Morbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6013-0072","authenticated-orcid":false,"given":"Katrin","family":"Hahn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,8,11]]},"reference":[{"issue":"7","key":"521_CR1","doi-asserted-by":"publisher","first-page":"387","DOI":"10.1038\/s41582-019-0210-4","volume":"15","author":"D Adams","year":"2019","unstructured":"Adams, D., Koike, H., Slama, M., & Coelho, T. 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Weiler reports advisory board or consultant honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, F. Hoffmann-La Roche, Novartis, Novo Nordisk, Pfizer, Purpose Pharma, and Swedish Orphan Biovitrum AB; speaker fees from Akcea Therapeutics, Alnylam Pharmaceuticals, AstraZeneca, and Biogen; and financial support for conference or meeting attendances from Akcea Therapeutics, Alnylam Pharmaceuticals, AstraZeneca, BridgeBio\/Eidos Therapeutics, F. Hoffmann-La Roche, Ionis Pharmaceuticals, and Pfizer; outside of the scope of the present work. MW serves as principal investigator in clinical trials sponsored by Alnylam Pharmaceuticals, BridgeBio\/Eidos Therapeutics, and Ionis Pharmaceuticals. MW is a member of the European Reference Network for Neuromuscular Diseases (ERN EURO-NMD), and a member of DGAK, DGN, and International Society of Amyloidosis (ISA).M. F. Dohrn reports advisory board or consultant honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals, Amicus, Applied Therapeutics, Pfizer Pharmaceuticals, Purpose Pharma, and Swedish Orphan Biovitrum AB; speaker fees from Akcea Therapeutics, Alnylam Pharmaceuticals, AstraZeneca, and Pfizer Pharmaceuticals; and financial support for conference or meeting attendances from Akcea Therapeutics, Alnylam Pharmaceuticals, AstraZeneca, Pfizer Pharmaceuticals, and Swedish Orphan Biovitrum AB; outside of the scope of the present work. MFD is a board member of the European CMT Research Association (ECRA), a member of the European Reference Network for Neuromuscular Diseases (ERN EURO-NMD), a board member of The Genesis Project (TGP) Foundation, a member of DGN, and DGAK. MFD is a member of the EAN\/PNS guideline task force on hereditary transthyretin amyloidosis with polyneuropathy.S. Gingele reports research support from Alnylam Pharmaceuticals, CSL Behring, Else Kr\u00f6ner Fresenius Foundation, Deutsche Forschungsgemeinschaft and Hannover Biomedical Research School (HBRS); financial reimbursement for consulting, advisory board activities, speaker fees and\/or contributions to congresses and travel support to attend scientific meetings from Alexion, Alnylam Pharmaceuticals, AstraZeneca, CSL Behring, GSK, Pfizer, Merck, Purpose Pharma and Takeda Pharmaceuticals all outside the submitted work. SG is a member of DGN and DGAK.T. Skripuletz received financial reimbursement for consulting, advisory board activities, speaker fees and\/or contributions to congresses and travel support to attend scientific meetings by Alexion, Alnylam, Amgen, argenx, Bayer, Biogen, BMS, Centogene, CSL Behring, Grifols, Hexal, Horizon, Janssen, Merck, Novartis, Pfizer, Purpose pharma, Roche, Sanofi, Siemens, SOBI, Takeda, Teva, Viatris. TS further received research support from Alnylam, CSL Behring, Merck, Novartis, Siemens.M. Schilling received advisory honoraria, speakers honoraria, and travel grants from Akcea, Alnylam, AstraZeneca, CSL Behring, Merck Healthcare, Pfizer, Sanofi Genzyme, Teva Pharmaceutical Industries.T. Hagenacker received financial reimbursement for consulting, advisory board activities, speaker fees and\/or contributions to congresses and travel support to attend scientific meetings by Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, Biogen, Hormosan, Novartis, Roche, Sanofi-Aventis, Purpose Pharma. TH further received research funding by Biogen, Novartis and Roche.E. Hund reports advisory board or consultant honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals, Pfizer Pharmaceuticals; speaker fees from Akcea Therapeutics, Alnylam Pharmaceuticals, AstraZeneca, and Pfizer Pharmaceuticals; and financial support for conference or meeting attendances from Akcea Therapeutics, Alnylam Pharmaceuticals and Pfizer Pharmaceuticals; outside of the scope of the present work.J. Sachau reports travel support from Alnylam Pharmaceuticals Inc. and Pfizer, consultant fees from Pfizer Pharma GmbH and AstraZeneca GmbH and speaker fees from Alnylam Germany GmbH, AstraZeneca and Gr\u00fcnenthal GmbH.R. Baron has received grants\/research support from Pfizer, Genzyme, Sanofi, Gr\u00fcnenthal, Mundipharma and Viatris. EU Project No 633491 DOLORisk. German Federal Ministry of Education and Research (BMBF): ERA-NET NEURON, IM-PAIN Project. German Research Network on Neuropathic Pain, NoPain system biology. German Research Foundation (DFG). He has received speaking fees Pfizer, Sanofi Aventis, Gr\u00fcnenthal, Eli Lilly, Desitin, Bayer, Janssen, Amicus, Alnylam, Novo Nordisk, Chiesi, Takeda, Stada, Ever, Unilab, Hexal, Viatris, AstraZeneca, AbbVie, Aristo, Esanum, Docflix, Vertanical, Denk. He has been a consultant for Pfizer, Sanofi Aventis, Gr\u00fcnenthal, Eli Lilly, AstraZeneca, Teva, AbbVie, Glenmark, Novartis, Biotest, Desitin, Bayer, Lateral, Alnylam, Confo, EVA, Merz, Neumentum, Algo, Nanobiotix, Chiesi, Stada, Viatris, Hexal, Heat2Move, Hoba, BioNTech, Esteve, Aristo, SIMR, Vertanical, SelectION, Max-Delbr\u00fcck-Centrum.H. F. Pernice has received personal funding, travel support, and\/or speaker fees by Alnylam Pharmaceuticals Inc, AstraZeneca, and Bayer Vital GmbH, and is part of the scientific advisory board for CURLIM. HP also received personal funding by Deutsche Gesellschaft f\u00fcr Muskelkranke (DGM) GmbH and Berlin Institute of Health (BIH). HFP is a board member of the European CMT Research Association (ECRA), and member of the Peripheral Nerve Society (PNS), DGN, and DGAK.S. Spethmann reports scientific grants from Innovation Committee at the Federal Joint Committee (G-BA), Pfizer Pharma GmbH, and Apple Inc.; Honoraria for lectures from Novartis Pharma GmbH, AstraZeneca GmbH, Pfizer Pharma GmbH, Alnylam Netherlands B.V., Boehringer Ingelheim Pharma GmbH, Medtronic GmbH, and Bayer Vital GmbH; travel fees from Pfizer Pharma GmbH, Alnylam Netherlands B.V., AstraZeneca GmbH, Medtronic GmbH, and Boehringer Ingelheim Pharma GmbH; Advisory fees from Boehringer Ingelheim Pharma GmbH, Novartis Pharma GmbH, Pfizer Pharma GmbH, Alnylam Netherlands B.V., and Bayer Vital GmbH.C. Khandanour received financial reimbursement for consulting, advisory board activities, speaker fees and\/or contributions to congresses and travel support to attend scientific meetings by Alnylam Pharmaceuticals Inc., AstraZeneca, Biogen, Novartis, Roche, Sanofi-Aventis, Johnson and Johnson, Amgen, Stemline, Alexion, Oncopeptides, BMS, TH further received research funding by Sanofi, MSD, Astra Zeneca.A. Carpinteiro reports consulting and\/or lecture fees from Alexion, Alnylam, Johnson & Johnson and Pfizer and travel and congress participation grants from Johnson & Johnson.C. Morbach received advisory honoraria, speakers honoraria, and travel grants from Alexion, Alnylam, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, Cytokinetics, Edwards, Eli Lilly, Intellia, Janssen, NovoNordisk, and Pfizer; she serves as principal investigator in trials sponsored by Alnylam, AstraZeneca, Bayer, Intellia, and NovoNordisk as well as a steering committee member in a trial sponsored by Alnylam.K. Hahn received financial reimbursement for consulting, advisory board activities, speaker fees and\/or contributions to congresses and travel support to attend scientific meetings by Akcea Therapeuticals Inc., Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, Bayer Vital GmbH, BridgeBio, GSK, Hormosan, Takeda Pharmaceutical Inc., Pfizer Pharmaceuticals Inc., Purpose Pharma, Swedish Orphan Biovitrum Inc and ViiV Healthcare GmbH. KH further received research funding by the foundation Charit\u00e9 (BIH clinical fellow and digital accelerator), Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, Hormosan and Pfizer Pharmaceuticals. KH is a board member of the European CMT Research Association (ECRA), a member of the European Reference Network for Neuromuscular Diseases (ERN EURO-NMD) a member of International Society of Amyloidosis (ISA), the Peripheral Nerve Society (PNS) and DGAK.","order":3,"name":"Ethics","label":"Competing interests","group":{"name":"EthicsHeading","label":"Declarations"}}],"article-number":"63"}}