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For the preceding 12\u00a0months, this compassionate use program (CUP) made risdiplam available to patients with SMA1\/2 in Germany who could not receive any approved SMA therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients and methods<\/jats:title><jats:p>Patients with SMA1\/2, aged\u2009\u2265\u20092\u00a0months at enrollment, could be included if they were not eligible for, no longer responsive to, or not able to tolerate nusinersen or not able to receive onasemnogene abeparvovec. Oral risdiplam dosing ranged from 0.2\u00a0mg\/kg to 5\u00a0mg depending on age and weight. All treatment decisions were made by the attending physicians, who were required to report all adverse events (AEs).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Between March 12, 2020 and March 30, 2021, 36 patients with SMA1 and 98\u00a0patients with SMA2 were enrolled, with 31 patients and 80 patients receiving \u2265\u20091 risdiplam dose, respectively. The median (range) age was 10.5 (3\u201352)\u00a0years in the SMA1 cohort, and 26.5 (3\u201360) years in the SMA2 cohort. 22.2% of patients with SMA1 and 48.0% with SMA2 were treatment-na\u00efve. Most patients were not eligible\/could not continue to receive nusinersen due to scoliosis\/safety risk (SMA1: 75.0%; SMA2: 96.9%), risks associated with sedation (77.8%; 63.3%), or loss of efficacy (30.6%; 12.2%). Safety data were generally in line with the safety profile of risdiplam in ongoing clinical studies. Gastrointestinal disorders were the most common AEs. For patients with SMA1, 30\u00a0AEs were reported in 13\u00a0cases with 2 serious AEs in 1\u00a0patient. For SMA2, 100\u00a0AEs were documented in 31\u00a0case reports, including 8 serious AEs in 2 patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>We present the first real-world safety data of risdiplam in patients with SMA in Germany. Our observations indicated no new safety signals under real-world conditions. Real-world SMA1\/2 populations comprise considerable numbers of patients who are not eligible for gene therapy and cannot tolerate or have failed nusinersen treatment. This medical need may be addressed by oral risdiplam.<\/jats:p><\/jats:sec>","DOI":"10.1186\/s13023-022-02420-8","type":"journal-article","created":{"date-parts":[[2022,7,19]],"date-time":"2022-07-19T11:06:59Z","timestamp":1658228819000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":32,"title":["Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany"],"prefix":"10.1186","volume":"17","author":[{"given":"Andreas","family":"Hahn","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ren\u00e9","family":"G\u00fcnther","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Albert","family":"Ludolph","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oliver","family":"Schwartz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Trollmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Weydt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathrin","family":"Neuland","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin Sebastian","family":"Schwaderer","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"}]},{"name":"the Risdiplam Compassionate Use Program Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2022,7,19]]},"reference":[{"issue":"1","key":"2420_CR1","doi-asserted-by":"publisher","first-page":"124","DOI":"10.1186\/s13023-017-0671-8","volume":"12","author":"IEC Verhaart","year":"2017","unstructured":"Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. 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All patients and\/or legally authorized representatives provided written informed consent to participate in the CUP.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable. All patients and\/or legally authorized representatives have signed a consent form in which they agreed to pseudonymized data being collected, analyzed and used in scientific publications.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"All authors received institutional support for their contribution to this CUP as well as medical writing support for publication of the data, both funded by Roche Pharma AG, Grenzach-Wyhlen, Germany. AH has received honoraria from Roche for participation at advisory boards and lectures. RG has received research grants from Biogen, personal fees and non-financial support from Biogen for lectures and for serving on advisory boards as well as personal fees from Hoffmann-La Roche for serving on advisory boards outside of the submitted work. AL has served on advisory boards for Roche Pharma AG. OS has declared no further competing interests. RT has received personal fees from Novartis, Sanofi, PTC, Biogen and Desitin for lectures and for serving on advisory boards outside of the submitted work. PW has received consulting fees from Roche, Biogen and ITF Pharma. MW advises for Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, Pfizer, Hoffmann-La Roche and Swedish Orphan Biovitrum, received lecture honoraria from Akcea Therapeutics, Alnylam Pharmaceuticals and Biogen, and received financial support for conference attendance from Biogen and Pfizer, all outside of the present work. KN and MSS are employees of Roche Pharma AG. TH has received research grants and personal fees for lectures from Biogen, Novartis Gene Therapies, Sanofi-Genzyme and Hoffmann-La Roche and for serving on advisory boards from Biogen, Sanofi-Genzyme, Hoffmann-La Roche, Alexion, Hormosan, Alnylam, Argenx and PTC outside the submitted work.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"276"}}