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Hoffmann-La Roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007013","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2023,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Risdiplam is an oral, survival of motor neuron\u00a02 (\n                    <jats:italic>SMN2<\/jats:italic>\n                    ) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA). SUNFISH (NCT02908685) Part 2, a Phase 3, randomized, double-blind, placebo-controlled study, investigated the efficacy and safety of risdiplam in type 2 and non\u2011ambulant type 3 SMA. The primary endpoint was met: a significantly greater change from baseline in 32-item Motor Function Measure (MFM32) total score was observed with risdiplam compared with placebo at month 12. After 12\u00a0months, all participants received risdiplam while preserving initial treatment blinding. We report 24-month efficacy and safety results in this population. Month 24 exploratory endpoints included change from baseline in MFM32 and safety. MFM\u2011derived results were compared with an external comparator. At month 24 of risdiplam treatment, 32% of patients demonstrated improvement (a change of\u2009\u2265\u20093) from baseline in MFM32 total score; 58% showed stabilization (a change of\u2009\u2265\u20090). Compared with an external comparator, a treatment difference of 3.12 (95% confidence interval [CI] 1.67\u20134.57) in favor of risdiplam was observed in MFM-derived scores. Overall, gains in motor function at month 12 were maintained or improved upon at month 24. In patients initially receiving placebo, MFM32 remained stable compared with baseline (0.31 [95% CI \u2013 0.65 to 1.28]) after 12\u00a0months of risdiplam; 16% of patients improved their score and 59% exhibited stabilization. The safety profile after 24\u00a0months was consistent with that observed after 12\u00a0months. Risdiplam over 24\u00a0months resulted in further improvement or stabilization in motor function, confirming the benefit of longer-term treatment.\n                  <\/jats:p>","DOI":"10.1007\/s00415-023-11560-1","type":"journal-article","created":{"date-parts":[[2023,2,3]],"date-time":"2023-02-03T11:03:59Z","timestamp":1675422239000},"page":"2531-2546","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":90,"title":["Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)"],"prefix":"10.1007","volume":"270","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1042-0108","authenticated-orcid":false,"given":"Maryam","family":"Oskoui","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0086-9529","authenticated-orcid":false,"given":"John 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Hoffmann-La Roche Ltd\u00a0during the conduct of the study and has received grants as a clinical trial investigator from Biogen. JWD has received fees for serving on scientific advisory boards from Biogen, Novartis, Sarepta\u00a0Therapeutics, and Avidity; has received consulting fees from Affinia Therapeutics and Shift Therapeutics for a therapeutic platform; and has received grant support for clinical trials from F. Hoffmann-La Roche\u00a0Ltd, Biogen, Novartis Gene Therapies, Cytokinetics, Scholar Rock, and Sarepta\u00a0Therapeutics. ND served on scientific advisory boards for F. Hoffmann-La Roche\u00a0Ltd, Biogen and Novartis Pharmaceuticals; he has received personal fees from Biogen and F. Hoffmann-La Roche\u00a0Ltd for congress and travel support. ESM reports that she has served on advisory boards for Biogen and Scholar Rock. She has received consulting fees, travel support, and speaker honoraria as an independent contractor from F. Hoffmann-La Roche\u00a0Ltd, Biogen, AveXis, and Scholar Rock. AN has received fees for serving on scientific advisory boards and speaker fees from F. Hoffmann-La Roche\u00a0Ltd. KS reports grants from F. Hoffmann-La Roche\u00a0Ltd\/Chugai Pharmaceutical during the conduct of the study and grants from Biogen Japan and lecture fees from Novartis Pharmaceuticals, outside the submitted work. CV reports personal fees and financial support to her institution from F. Hoffmann-La Roche\u00a0Ltd for activities outside the submitted work. GB received consultancy fees and speaker honoraria from F. Hoffmann-La Roche\u00a0Ltd, AveXis\/Novartis Gene Therapy, and Biogen, and grants from\u00a0F. Hoffmann-La Roche\u00a0Ltd. NG reports fees for serving on advisory boards and presentations at symposia from F. Hoffmann-La Roche\u00a0Ltd, Biogen, AveXis, and Novartis. JK received grants or contracts from Novartis Gene Therapies and Biogen. He has received consulting and speaker fees from F. Hoffmann La Roche\u00a0Ltd, Biogen, and Novartis Gene Therapies and consulting fees from Scholar Rock. AK-P reports that she received an institutional support grant from Biogen; serving on scientific advisory boards for and receiving speaker honoraria from Biogen, F. Hoffmann-La Roche\u00a0Ltd, Novartis, and PTC Therapeutics; and receiving personal fees from Biogen and F. Hoffmann-La Roche\u00a0Ltd for travel support. LS received grants and personal fees from F. Hoffmann-La Roche\u00a0Ltd, Biogen, and AveXis\/Novartis Gene Therapies and personal fees from Cytokinetics, BioHaven, and Scholar Rock, outside the submitted work. GP, KG, HK, CM, TM, RSS, HS, WYY, and PF report that they are current employees\u00a0of and stockholders in F. Hoffmann-La Roche\u00a0Ltd. EM has received fees for serving on scientific advisory boards; speaker fees from F. Hoffmann-La Roche\u00a0Ltd, Biogen, AveXis\/Novartis, Scholar Rock, and Cytokinetics; and grants from Biogen during the conduct of the study.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"This trial was approved by an ethics committee at each study site and was conducted in accordance with Good Clinical Practice guidelines and with the World Medical Association Declaration of Helsinki.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}},{"value":"Written informed consent was provided by the patient or by the patients\u2019 legally authorized representative before participating in the study.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed consent"}}]}}