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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
David Adams, Michael Polydefkis, Alejandra González‐Duarte, Jonas Wixner, Arnt V. Kristen, Hartmut Schmidt, John L. Berk, Inés Asunción Losada López, Angela Dispenzieri, Dianna Quan, Isabel Conceição, Michel Slama, Julian D. Gillmore, Theodoros Kyriakides, Senda Ajroud‐Driss, Márcia Waddington‐Cruz, Michelle M. Mezei, Violaine Planté‐Bordeneuve, Shahram Attarian, Elizabeth A. Mauricio, Thomas H. Brannagan, Mitsuharu Ueda, Emre Aldinc, Jing Jing Wang, Matthew T. White, Erhan Berber, Marianne T. Sweetser, Teresa Coelho, Giuseppe Vita, Vincenzo Rizzo, Massimo Russo, Anna Mazzeo, Luca Gentile, Caitlin Brueckner, Victoria Lazzari, Janice F. Wiesman, Douglas DeLong, Jennifer Victory, James A. R. Dalton, John J. May, Catherine Gilmore, Saran Diallo, Émilien Delmont, Jean Pouget, Annie Verschueren, Aude‐Marie Grapperon, Emmanuelle Salort‐Campana, Ana Tarina Alvarez Lopes, Filipa Lamas, Carlos Marques‐Neves, José Castro, Pedro Pereira, Isabel Castro, Ana Franco, Miguel Oliveira Santos, Conceição de Azevedo Coutinho, Catarina Falcão de Campos, Antonio Hipólito Reis, Nuno Correia, Javier Martínez Pérez, Ana Silva, Cristina Alves, Márcio Cardoso, Kátia Valdrez, Julia R Monte, Bernardete Pessoa, Nádia Guimarães, Mónica Freitas, Joana Ramalho, Natália Ferreira, Daisuke Kuzume, Céline Tard, Nawal Waucquier, Isabelle Rougeaux, Sylvie Brice, Emmanuelle Kasprzyk, Elise Elrezzi, Sayah Meguig, É. Hachulla, Clément Gauvain, Maria-Claire Migaud-Chervy, Dominique Deplanque, Elsa Jozefowicz, Loïc Lebellec, David B. 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Weimer, Comana Cioroiu, Sakis Lambrianides, Rana Abu-Manneh, Eleni Zamba‐Papanicolaou, Petros Agathangelou, Eleni Leonidou, Satoshi Tada, Akemi Fujita, Masahiro Nagai, Rina Ando, Yuko Hosokawa, Yuki Yamanishi, J. Scott Overcash, Elena Giardino, Leslie Boyer, Lien Dang, An K. Le, Tyler Nguyen, Lien Giang, P Sellers, Leyla Tran, Nghi Truong, Maita Vinas, Nicole Hrkman, S. David Miller, David Nguyen, Ashley Smith, Helen Pu, S. Li, Thao Vuong, Holly Dioso, Sinikka Green, Kia Lee, Hanh Chu, Michael R. Waters, Derya J Coskun, Karla A Zepeda, William O’Riordan, Laura Obici, Andrea Cortese, Alessandro Lozza, Giampaolo Merlini, Vittorio Rosti, Mario Sabatelli, Giulia Bisogni, Daniela Bernardo, Marco Luigetti, Andrea Di Paolantonio, Valeria Guglielmino, Ângela Romano, Hans Nienhuis, Janita Bulthuis-Kuiper, Olga Gerk, Hannah Ulbricht, Lenka A. 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Bibliographic record
VenueThe Lancet Neurology · 2020
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicAmyloidosis: Diagnosis, Treatment, Outcomes
Canadian institutionsUniversity of British Columbia
FundersAgence Nationale de la RechercheAlnylam Pharmaceuticals
KeywordsMedicineTransthyretinPolyneuropathyClinical trialAmyloidosisInternal medicinePlaceboPediatricsPhysical therapyPathologyAlternative medicine
Abstract
No abstract in any covered source. Its absence is recorded, not treated as a negative.
No abstract. This is not a gap in this database; OpenAlex has none either. 23.3% of the frame is in this state, and the screen finds HALF as much metaresearch here, so the absence is a measured bias rather than a missing field.
How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.369
Threshold uncertainty score0.723
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
Opus teacher head0.037
GPT teacher head0.293
Teacher spread0.256 · how far apart the two teachers sit on this one work
Validation status
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".
Quick stats
Citations156
Published2020
Admission routes1
Has abstractno