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Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
Eugenio Mercuri, Nicolas Deconinck, Elena Mazzone, A. Nascimento, Maryam Oskoui, Kayoko Saito, Carole Vuillerot, Giovanni Baranello, Odile Boespflug‐Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera‐Pruszczyk, Laurent Servais, Marianne Gerber, Ksenija Gorni, Omar Khwaja, Heidemarie Kletzl, R. Scalco, Hannah Staunton, Wai Yin Yeung, Paulo Fontoura, Joseph J. Volpe, John Posner, Ulrich Kellner, Rosaline C. M. Quinlivan, Aurore Daron, Stéphanie Delstanche, Romain Bruninx, Fabian Dal Farra, O. Schneider, Irina Balikova, Patricia Delbeke, Inge Joniau, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Ingele Casteels, Liesbeth De Waele, Cathérine Cassiman, Lies Prové, David Kinoo, Lisa Vancampenhout, M. van den Hauwe, Annelies Van Impe, Alexandra Prufer de Queiroz Campos Araújo, Aline Chacon Pereira, Flávia Nardes, Lorena Haefeli, Júlia Dutra Rossetto, Marcos Ferreira Rebel, Jaqueline Almeida Pereira, Craig Campbell, Sapna Sharan, Wendy L. McDonald, Cheryl Scholtes, Jean K. Mah, Maria Sframeli, Angela W. Chiu, Jane Hagel, Raquel Beneish, Gaela Cariou-Palmer, Connie Pham, Daniela Toffoli, Stéphanie Arpin, Sarah Turgeon Desilets, Yi Wang, Chaoping Hu, Jianfeng Huan, Qian Chen, Li Shen, Ying Xiao, Zhenxuan Zhou, Hui Li, Sujuan Wang, Hui Xiong, Xingzhi Chang, Hui Dong, Ying Liu, Tian Sang, Cuijie Wei, Jing Wen, Yiwen Cao, Xingyao Ly, Jingjing Zhao, Wenzhu Li, Lun Qin, Nina Barišić, Martina Galiot Delić, Petra Kristina Ivkić, Nenad Vukojević, Ivana Kern, B Najdanović, Marin Skugor, Teresa Gidaro, A. Seferian, Silvana De Lucia, Emmanuel Barreau, Nabila Mnafek, Marta Momtchilova, Helene Peche, Carole Valherie, Allison Grange, Charlotte Lilien, Darko Milaščević, Shotaro Tachibana, Claudia Ravelli, Ruxandra Cardaş, Jessica Taytard, Guillaume Aubertin, Laura Vanden Brande, Jean‐Baptiste Davion, Stéphanie Coopman, Ikram Bouacha, Philippe Debruyne, Sabine Defoort, Gilles Derlyn, Florian Leroy, Loïc Danjoux, J Guilbaud, Isabelle Desguerre, Christine Barnérias, Michaëla Semeraro, Dominique Brémond‐Gignac, Lenaïc Bruère, Maxence Rateaux, Élodie Deladrière, Virginie Germa, Yann Péréon, Sandra Mercie, Fanny Billaud, Lucie Le Goff, Guy Letellier, Aurélie Portefaix, Camille De-Montferrand, Laure Le-Goff, Stéphanie Fontaine, Manel Saidi, Aurélie Barrière, Marie Tinat, Michelle Dreesbach, Wolf A. Lagrèze, Bettina Michaelis, Fanni Molnár, Dorina Seger, Sibylle Emilie Vogt, Enrico Bertini, Adele D’Amico, Sergio Petroni, Anna Maria Bonetti, Adelina Carlesi, Irene Mizzoni, Claudio Bruno, Enrico Priolo, Giuseppe Rao, Simone Morando, Paola Tacchetti, A. Zuffi, Giacomo P. Comi, Roberta Brusa, Stefania Corti, Velardo Daniele, Alessandra Govoni, Francesca Magri, Valeria Minorini, Silvia Osnaghi, Francesca Abbati, Federica Fassini, Michaela Foà, Amaqlia Lopopolo, Megi Meneri, Francesca Zoppas, Valeria Parente, Riccardo Masson, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Maria Teresa Arnoldi, M. E. F Viganó, Riccardo Zanin, Laura Antonaci, Roberto De Sanctis, Marika Pane, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, G. D'Amico, Lorenzo Orazi, Giorgia Coratti, Kazuhiro Haginoya, Atsuko Kato, Yuko Morishita, Ryutaro Kira, Kiyomu Akiyama, Miwako Goto, Yujiro Mori, Misato Okamoto, Saki Tsutsui, Yuta Takatsuji, Aya Tanaka, Hirofumi Komaki, Miina Omori, Ippei Suzuki, Mizuki Takeuchi, Daisuke Todoroki, Seji Watanabe, Tomoko Matsubayashi, Emi Inakazu, Hiroe Nagura, Akira Suzuki, Manami Usui, Nobutsune Ishikawa, Yousuke Harada, Kazuhiko Hirata, Kana Michiue, Kazuyuki Ueda, Junko Fujitani, Reiko Arakawa, Kozue Takano, Shigeko Yashiro, Maiko Seki, Nozomi Sano, Koji Fukuyama, Yuki Matsumoto, Hirofumi Miyazaki, Minoru Shibata, Kyoko Kobayashi, Yukie Nakamura, Yasuhiro Takeshima, Moe Kuma, Anna Frączek, Maria Jędrzejowska, Anna Łusakowska, Agnieszka Czeszyk-Piotrowicz, Wojciech Hautz, Klaudia Rakusiewicz, Małgorzata Burlewicz, Zuzanna Gierlak-Wojcicka, Malwina Kępa, Adam Sikorski, Marcin Sobieraj, Maria Mazurkiewicz-Bełdzińska, Anna Lemska, Sandra Modrzejewska, Mateusz Koberda, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Barbara Steinborn, Magdalena Dalz, J Grabowska, Wojciech Hajduk, Justyna Janasiewicz-Karachitos, Monika Klimas, Marcin Stopa, Ewa Gajewska, Beata Pusz, D. Vlodavets, Evgenia Melnik, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Vedrana Milić Rašić, V. Branković, Ana Kosać, Olivera Djokić, Vesna Jakšić, Ana Pepić, Jelena Martinović, Eduardo F. Tizzano, Nieves Martín Begué, Charlotte Wolley-Dod, Olaia Subirà, Bernat Planas Pascual, Esther Toro‐Tamargo, Marcos Madruga Garrido, José David Medina Romero, M Salinas, A. Nascimento Osorio, Ana Díaz Cortés, Enrique Jiménez Gañan, Simone Dowon Suh, Julita Medina Cantillo, O. Moya, Nuria Padros, Sandra Roca Urraca, Hugo Gonzalez Valdivia, Samuel Ignacio Pascual Pascual, Sofia Manuel, Susana Noval Martín, Paul Burnham, Sandra Espinosa, M. Martínez Moreno, Haluk Topaloğlu, İbrahim Öncel, Nesibe Eroglu Ertugru, Bahadır Konuşkan, Bora Eldem, Sibel Kadayıfçılar, İ. Alemdaroğlu, Aynur Ayşe Karaduman, O. Yılmaz, Neslihan Bilgin, Seher Sari, Claudia A. Chiriboga, John J. Lee, Donnielle Rome-Martin, Shannon Beres, Tina Duong, Richard Gee, Sally Dunaway Young, Sabine Fuerst‐Recktenwald, Anne Marquet, Nicoletta Milani Muelhardt, Dylan Trundell
Bibliographic record
VenueThe Lancet Neurology · 2021
Typeletter
Languageen
FieldMedicine
TopicNeurogenetic and Muscular Disorders Research
Canadian institutionsMcGill University
FundersRoche
KeywordsMedicineSpinal muscular atrophyPlaceboClinical endpointPhysical therapyAtrophyClinical trialAdverse effectInternal medicineDiseasePathology
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 machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.002
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Randomized trial · Consensus signal: Randomized trial
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.007
Threshold uncertainty score0.022
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.003 | 0.002 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.004 | 0.003 |
| Insufficient payload (model declined to judge) | 0.007 | 0.001 |
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 head0.046
GPT teacher head0.337
Teacher spread0.291 · 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 source (direct Gemma or distilled Codex), not a consensus.
The models applied no category: nothing in the taxonomy fit this work.
Study designRandomized trial
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
Citations214
Published2021
Admission routes1
Has abstractno