{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T02:09:29Z","timestamp":1792634969802,"version":"4.5.0"},"reference-count":44,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T00:00:00Z","timestamp":1746489600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T00:00:00Z","timestamp":1746489600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Orphanet J Rare Dis"],"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:sec>\n            <jats:title>Background<\/jats:title>\n            <jats:p>Onasemnogene abeparvovec (OA) is an adeno-associated virus vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy(SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene. This study focused on analysis of the postmarketing adverse events(AEs) of onasemnogene abeparvovec (OA) reported in the US Food and Drug Administration public data open project (openFDA) database to assess the safety of OA in the real world and to provide a reference for the rational use of this drug in the clinic.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results<\/jats:title>\n            <jats:p>In total, 1,959 AEs were reported with \u201conasemnogene abeparvovec\u201d as the primary suspected drug. The top 5 most frequent AEs were pyrexia (461 cases), vomiting (434 cases), aspartate aminotransferase increase (284 cases), alanine aminotransferase increase (260 cases), and hepatic enzyme increase (237 cases). A total of 77 alert signals were generated, 60 of which were not included in the drug label. The top 5 signals included troponin I increase ( ROR of 895.21, 95% CI: 734.43-1091.18), troponin T increase ( ROR of 313.30, 95% CI:220.85-444.44), rhinovirus infection ( ROR of 175.80, 95% CI:130.86-236.17), troponin increase ( ROR of 143.49, 95% CI:114.96\u2013179.10), and increased bronchial secretion ( ROR of 142.71, 95% CI:96.63-210.77). Further analysis of AEs associated with gender and age differences identified 14 high-risk signals related to gender and 10 high-risk signals related to age. Female patients should be vigilant for vomiting, thrombotic microangiopathy, increased troponin T, proteinuria, haematuria, haemolytic anaemia, urinary tract infection, generalised oedema, and atypical haemolytic uraemic syndrome. Male patients should be alert to increased hepatic enzyme, increased bronchial secretion, respiratory tract infection, pallor, and increased blood creatine phosphokinase MB. Patients under 2 years of age should be vigilant for lethargy, increased monocyte count, decreased blood creatinine, and decreased neutrophil count. Patients over 2 years of age should be alert to hypertension, haematuria, rhinovirus infection, increased blood creatine phosphokinase, headache, and malaise.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusions<\/jats:title>\n            <jats:p>Mining of OA alert signals using the openFDA database provides supplementary information on AEs not included in the drug label. Clinical attention should be focused on common, strong-signal, and label-unmentioned AEs to optimise medication regimens and control risks in clinical use.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1186\/s13023-025-03715-2","type":"journal-article","created":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T12:55:43Z","timestamp":1746536143000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study"],"prefix":"10.1186","volume":"20","author":[{"ORCID":"https:\/\/orcid.org\/0009-0000-4930-7685","authenticated-orcid":false,"given":"Tianyu","family":"Chen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiying","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jingfang","family":"Ye","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuzhu","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ting","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuezhen","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,5,6]]},"reference":[{"issue":"1","key":"3715_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. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review. Orphanet J Rare Dis. 2017;12(1):124. https:\/\/doi.org\/10.1186\/s13023-017-0671-8","journal-title":"Orphanet J Rare Dis"},{"issue":"1","key":"3715_CR2","doi-asserted-by":"publisher","first-page":"52","DOI":"10.1038\/s41572-022-00380-8","volume":"8","author":"E Mercuri","year":"2022","unstructured":"Mercuri E, Sumner CJ, Muntoni F, Darras BT, Finkel RS. Spinal muscular atrophy. Nat Rev Dis Primers. 2022;8(1):52. https:\/\/doi.org\/10.1038\/s41572-022-00380-8","journal-title":"Nat Rev Dis Primers"},{"issue":"10","key":"3715_CR3","doi-asserted-by":"publisher","first-page":"1446","DOI":"10.3390\/brainsci13101446","volume":"13","author":"S Crisafulli","year":"2023","unstructured":"Crisafulli S, Boccanegra B, Vitturi G, Trifir\u00f2 G, De Luca A. Pharmacological therapies of spinal muscular atrophy: a narrative review of preclinical, clinical-experimental, and real-world evidence. Brain Sci. 2023;13(10):1446. https:\/\/doi.org\/10.3390\/brainsci13101446","journal-title":"Brain Sci"},{"key":"3715_CR4","doi-asserted-by":"publisher","first-page":"890860","DOI":"10.3389\/fneur.2022.890860","volume":"13","author":"K Kotulska","year":"2022","unstructured":"Kotulska K, Jozwiak S, Jedrzejowska M, Gos M, Ogrodnik M, et al. Newborn screening and gene therapy in SMA: challenges related to vaccinations. Front Neurol. 2022;13:890860. https:\/\/doi.org\/10.3389\/fneur.2022.890860","journal-title":"Front Neurol"},{"issue":"7","key":"3715_CR5","doi-asserted-by":"publisher","first-page":"511","DOI":"10.1136\/archdischild-2021-323605","volume":"108","author":"VL Gowda","year":"2022","unstructured":"Gowda VL, Fernandez-Garcia MA, Jungbluth H, Wraige E. New treatments in spinal muscular atrophy. Arch Dis Child. 2022;108(7):511\u20137. https:\/\/doi.org\/10.1136\/archdischild-2021-323605","journal-title":"Arch Dis Child"},{"issue":"3","key":"3715_CR6","doi-asserted-by":"publisher","first-page":"e36197","DOI":"10.7759\/cureus.36197","volume":"15","author":"T Ogbonmide","year":"2023","unstructured":"Ogbonmide T, Rathore R, Rangrej SB, Hutchinson S, Lewis M, Ojilere S, et al. Gene therapy for spinal muscular atrophy (SMA): a review of current challenges and safety considerations for onasemnogene abeparvovec (Zolgensma). Cureus. 2023;15(3):e36197. https:\/\/doi.org\/10.7759\/cureus.36197","journal-title":"Cureus"},{"key":"3715_CR7","unstructured":"Initial US, Approval. Food and Drug Administration. ZOLGENSMA\u00ae (onasemnogene abeparvovec-xioi) suspension, for intravenous infusion. PRESCRIBING INFORMATION; 2019. https:\/\/www.novartis.us\/sites\/www.novartis.us\/files\/zolgensma.pdf. Revised: 10\/2021."},{"key":"3715_CR8","unstructured":"European Medicines Agency. Zolgensma (onasemnogene abeparvovec). https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/zolgensma"},{"key":"3715_CR9","doi-asserted-by":"publisher","first-page":"7341","DOI":"10.2147\/IJGM.S369021","volume":"15","author":"L Ramos-Platt","year":"2022","unstructured":"Ramos-Platt L, Elman L, Shieh PB. Experience and perspectives in the US on the evolving treatment landscape in spinal muscular atrophy. Int J Gen Med. 2022;15:7341\u201353. https:\/\/doi.org\/10.2147\/IJGM.S369021","journal-title":"Int J Gen Med"},{"key":"3715_CR10","doi-asserted-by":"publisher","first-page":"592","DOI":"10.1038\/s41434-022-00341-6","volume":"30","author":"I Bitetti","year":"2023","unstructured":"Bitetti I, Lanzara V, Margiotta G, Varone A. Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study. Gene Ther. 2023;30:592\u20137. https:\/\/doi.org\/10.1038\/s41434-022-00341-6","journal-title":"Gene Ther"},{"key":"3715_CR11","doi-asserted-by":"publisher","first-page":"383","DOI":"10.13748\/j.cnki.issn","volume":"40","author":"C Zhao","year":"2023","unstructured":"Zhao C, Zhang DX, Wang J. Data mining and analysis of adverse events of interleukin-17A inhibitors in the aspects of inflammatory bowel disease based on FAERS. Chin J Mod Appl Pharm. 2023;40:383\u20137. https:\/\/doi.org\/10.13748\/j.cnki.issn","journal-title":"Chin J Mod Appl Pharm"},{"key":"3715_CR12","unstructured":"European Medicine Agency. New gene therapy to treat spinal muscolar atrophy; European Medicine Agency: Amsterdam, The Netherlands. 2020; Volume 31."},{"key":"3715_CR13","unstructured":"European Medicines Agency (EMA). Summary of the risk management for Zolgensma\u00ae (onasemnogene abeparvovec). Last updated:18\/10\/2023. Available online: https:\/\/www.ema.europa.eu\/en\/documents\/rmp-summary\/zolgensma-epar-risk-management-plan-summary_en.pdf"},{"key":"3715_CR14","doi-asserted-by":"publisher","first-page":"689","DOI":"10.1007\/s40264-016-0413-1","volume":"39","author":"F Montastruc","year":"2016","unstructured":"Montastruc F, Salvo F, Arnaud M, B\u00e9gaud B, Pariente A. Signal of gastrointestinal congenital malformations with antipsychotics after minimising competition bias: a disproportionality analysis using data from vigibase. Drug Saf. 2016;39:689\u201396. https:\/\/doi.org\/10.1007\/s40264-016-0413-1","journal-title":"Drug Saf"},{"issue":"1","key":"3715_CR15","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1248\/yakushi","volume":"137","author":"E Kose","year":"2017","unstructured":"Kose E, Uno K, Hayashi H. Evaluation of the expression profile of extrapyramidal symptoms due to antipsychotics by data mining of Japanese adverse drug event report (Jader) database. Yakugaku Zasshi. 2017;137(1):111\u201320. 10.1248 \/yakushi.","journal-title":"Yakugaku Zasshi"},{"key":"3715_CR16","unstructured":"FDA. OpenFDA\/download [EB\/OL]. [cited 2023 Feb 17].Available from:https:\/\/open.fda.gov\/downloads\/2023"},{"key":"3715_CR17","doi-asserted-by":"crossref","first-page":"237","DOI":"10.3113\/JSOA.2022.0237","volume":"31","author":"G Provenzano","year":"2022","unstructured":"Provenzano G, Provenzano D, Best BJ, Afsari A. Categorization of adverse events reported to the FDA pertaining to tibia intramedullary nailing. J Surg Orthop Adv. 2022;31:237\u201341. PMID: 36594981.","journal-title":"J Surg Orthop Adv"},{"issue":"11","key":"3715_CR18","doi-asserted-by":"publisher","first-page":"1505","DOI":"10.1080\/14740338.2020.1799975","volume":"19","author":"L Peng","year":"2020","unstructured":"Peng L, Xiao K, Ottaviani S, Stebbing J, Wang YJ. A real-world disproportionality analysis of FDA adverse event reporting system (FAERS) events for baricitinib. Expert Opin Drug Saf. 2020;19(11):1505\u201311. https:\/\/doi.org\/10.1080\/14740338.2020.1799975","journal-title":"Expert Opin Drug Saf"},{"issue":"3","key":"3715_CR19","doi-asserted-by":"publisher","first-page":"596","DOI":"10.1093\/jamia\/ocv153","volume":"23","author":"TA Kass-Hout","year":"2016","unstructured":"Kass-Hout TA, Xu ZH, Mohebbi M, Nelsen H, Baker A, et al. OpenFDA: an innovative platform providing access to a wealth of FDA\u2019s publicly available data. J Am Med Informn Assoc. 2016;23(3):596\u2013600. https:\/\/doi.org\/10.1093\/jamia\/ocv153","journal-title":"J Am Med Informn Assoc"},{"issue":"11","key":"3715_CR20","doi-asserted-by":"publisher","first-page":"1255","DOI":"10.1007\/s40265-019-01162-5","volume":"79","author":"SM Hoy","year":"2019","unstructured":"Hoy SM. Onasemnogene abeparvovec: first global approval. Drugs. 2019;79(11):1255\u201362. https:\/\/doi.org\/10.1007\/s40265-019-01162-5","journal-title":"Drugs"},{"key":"3715_CR21","unstructured":"The US. Food and Drug Administration. Package Insert - ZOLGENSMA. [cited 2023 Feb 17].Available from:https:\/\/www.fda.gov\/media\/126109\/download"},{"key":"3715_CR22","unstructured":"Novartis Pharmaceuticals(. 2019). AveXis receives FDA approval for Zolgensma\u00ae, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA). [cited 2019 May 24].Available from:https:\/\/www.novartis.com\/us-en\/news\/media-releases\/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma"},{"issue":"3","key":"3715_CR23","doi-asserted-by":"publisher","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","volume":"74","author":"D Chand","year":"2021","unstructured":"Chand D, Mohr F, McMillan H, Tukov FF, Montgomery K, et al. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. J Hepatol. 2021;74(3):560\u20136. 10.1016\/ j.jhep.2020.11.001.","journal-title":"J Hepatol"},{"issue":"12","key":"3715_CR24","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1007\/s40261-023-01320-4","volume":"43","author":"W Zhuang","year":"2023","unstructured":"Zhuang W, Lu M, Wu Y, Chen Z, Wang M, Wang X, et al. Safety concerns with nusinersen, risdiplam, and onasemnogene abeparvovec in spinal muscular atrophy: a real-world pharmacovigilance study. Clin Drug Investig. 2023;43(12):949\u201362. https:\/\/doi.org\/10.1007\/s40261-023-01320-4","journal-title":"Clin Drug Investig"},{"key":"3715_CR25","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0157753","author":"R B\u00f6hm","year":"2016","unstructured":"B\u00f6hm R, von Hehn L, Herdegen T, Klein HJ, Bruhn O, Petri H, et al. OpenVigil FDA - Inspection of U.S. American Adverse Drug Events Pharmacovigilance Data and Novel Clinical Applications. PLoS ONE. 2016;11(6):e0157753. https:\/\/doi.org\/10.1371\/journal.pone.0157753"},{"issue":"11","key":"3715_CR26","doi-asserted-by":"publisher","first-page":"804","DOI":"10.1038\/d41573-021-00164-x","volume":"20","author":"A Mullard","year":"2021","unstructured":"Mullard A. Gene therapy community grapples with toxicity issues, as pipeline matures. Nat Rev Drug Discov. 2021;20(11):804\u20135. https:\/\/doi.org\/10.1038\/d41573-021-00164-x","journal-title":"Nat Rev Drug Discov"},{"issue":"1","key":"3715_CR27","doi-asserted-by":"publisher","first-page":"47","DOI":"10.1038\/s41587-020-0741-7","volume":"39","author":"GN Nguyen","year":"2021","unstructured":"Nguyen GN, Everett JK, Kafle S, Roche AM, Raymond HE, Leiby J, et al. A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells. Nat Biotechnol. 2021;39(1):47\u201355. https:\/\/doi.org\/10.1038\/s41587-020-0741-7","journal-title":"Nat Biotechnol"},{"issue":"7","key":"3715_CR28","doi-asserted-by":"publisher","first-page":"1390","DOI":"10.1038\/s41591-022-01867-3","volume":"28","author":"KA Strauss","year":"2022","unstructured":"Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, et al. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the phase III SPR1NT trial. Nat Med. 2022;28(7):1390\u20137. https:\/\/doi.org\/10.1038\/s41591-022-01867-3","journal-title":"Nat Med"},{"issue":"1","key":"3715_CR29","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1038\/s41591-022-01867-3","volume":"6","author":"C Wei\u00df","year":"2022","unstructured":"Wei\u00df C, Ziegler A, Becker L, Johannsen J, Brennenstuhl H, et al. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and body weight up to 15 kg: an observational cohort study. Lancet Child Adolesc Health. 2022;6(1):17\u201327. https:\/\/doi.org\/10.1038\/s41591-022-01867-3","journal-title":"Lancet Child Adolesc Health"},{"issue":"9","key":"3715_CR30","doi-asserted-by":"publisher","first-page":"685","DOI":"10.1038\/s41434-023-00401-5","volume":"30","author":"DH Chand","year":"2023","unstructured":"Chand DH, Sun R, Diab KA, Kenny D, Tukov FF. Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings. Gene Ther. 2023;30(9):685\u201397. https:\/\/doi.org\/10.1038\/s41434-023-00401-5","journal-title":"Gene Ther"},{"issue":"1","key":"3715_CR31","doi-asserted-by":"publisher","first-page":"67","DOI":"10.1186\/s13023-017-0613-5","volume":"12","author":"CA Wijngaarde","year":"2017","unstructured":"Wijngaarde CA, Blank AC, Stam M, Wadman RI, van den Berg LH, van der Pol WL. Cardiac pathology in spinal muscular atrophy: a systematic review. Orphanet J Rare Dis. 2017;12(1):67. https:\/\/doi.org\/10.1186\/s13023-017-0613-5","journal-title":"Orphanet J Rare Dis"},{"key":"3715_CR32","doi-asserted-by":"publisher","first-page":"265","DOI":"10.1016\/j.jpeds.2020.11.054","volume":"231","author":"DH Chand","year":"2021","unstructured":"Chand DH, Zaidman C, Arya K, Millner R, Farrar MA, et al. Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series. J Pediatr. 2021;231:265\u20138. https:\/\/doi.org\/10.1016\/j.jpeds.2020.11.054","journal-title":"J Pediatr"},{"issue":"Supplement 15","key":"3715_CR33","doi-asserted-by":"publisher","first-page":"5483","DOI":"10.1212\/WNL.94.15_supplement.5483","volume":"94","author":"N Prabhu","year":"2020","unstructured":"Prabhu N, Saylam E, Louis C, Moss M, Millner R, et al. Thrombotic microangiopathy (TMA): a potential adverse reaction post onasemnogene abeparvovec (onasemnogene abeparvovec-xioi) therapy for spinal muscular atrophy (SMA). Neurology. 2020;94(Supplement 15):5483.","journal-title":"Neurology"},{"issue":"3","key":"3715_CR34","doi-asserted-by":"publisher","first-page":"339","DOI":"10.1002\/acn3.51519","volume":"9","author":"AM D\u2019Silva","year":"2022","unstructured":"D\u2019Silva AM, Holland S, Kariyawasam D, Herbert K, Barclay P, et al. Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy. Ann Clin Transl Neurol. 2022;9(3):339\u201350. https:\/\/doi.org\/10.1002\/acn3.51519","journal-title":"Ann Clin Transl Neurol"},{"issue":"1","key":"3715_CR35","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1055\/a-1538-4936","volume":"234","author":"D Witte","year":"2022","unstructured":"Witte D, Hartmann H, Drube J, Haffner D, Illsinger S. Thrombotic microangiopathy (TMA) after gene replacemant therapy (GRT) due to spinal muscular atrophy: case summary and recommendations for treatment. Klin Padiatr. 2022;234(1):42\u20137. https:\/\/doi.org\/10.1055\/a-1538-4936","journal-title":"Klin Padiatr"},{"issue":"9","key":"3715_CR36","doi-asserted-by":"publisher","first-page":"1478","DOI":"10.1111\/jcpt.13733","volume":"47","author":"F Galletta","year":"2022","unstructured":"Galletta F, Cucinotta U, Marseglia L, Cacciola A, Gallizzi R, et al. Hemophagocytic lymphohistiocytosis following gene replacement therapy in a child with type 1 spinal muscular atrophy. J Clin Pharm Ther. 2022;47(9):1478\u201381. https:\/\/doi.org\/10.1111\/jcpt.13733","journal-title":"J Clin Pharm Ther"},{"issue":"4","key":"3715_CR37","doi-asserted-by":"publisher","first-page":"284","DOI":"10.1016\/S1474-4422(21)00001-6","volume":"20","author":"JW Day","year":"2021","unstructured":"Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford O, Darras BT, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(4):284\u201393. https:\/\/doi.org\/10.1016\/S1474-4422(21)00001-6","journal-title":"Lancet Neurol"},{"issue":"3","key":"3715_CR38","doi-asserted-by":"publisher","first-page":"431","DOI":"10.1111\/jpc.16340","volume":"59","author":"D Yang","year":"2023","unstructured":"Yang D, Ruan Y, Chen Y. Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: a systematic review and meta-analysis. J Paediatr Child Health. 2023;59(3):431\u20138. https:\/\/doi.org\/10.1111\/jpc.16340","journal-title":"J Paediatr Child Health"},{"issue":"4","key":"3715_CR39","doi-asserted-by":"publisher","first-page":"413","DOI":"10.1002\/mus.27363","volume":"64","author":"EA Kichula","year":"2021","unstructured":"Kichula EA, Proud CM, Farrar MA, Kwon JM, Saito K, Desguerre I, et al. Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy. Muscle Nerve. 2021;64(4):413\u201327. https:\/\/doi.org\/10.1002\/mus.27363","journal-title":"Muscle Nerve"},{"key":"3715_CR40","doi-asserted-by":"publisher","first-page":"740","DOI":"10.1089\/hum.2021.255","volume":"33","author":"FF Tukov","year":"2022","unstructured":"Tukov FF, Mansfield K, Milton M, Meseck E, Penraat K, et al. Single-dose intrathecal dorsal root ganglia toxicity of onasemnogene abeparvovec in cynomolgus monkeys. Hum Gene Ther. 2022;33:740\u201356. https:\/\/doi.org\/10.1089\/hum.2021.255","journal-title":"Hum Gene Ther"},{"issue":"7","key":"3715_CR41","doi-asserted-by":"publisher","first-page":"1390","DOI":"10.1038\/s41591-022-01867-3","volume":"28","author":"KA Strauss","year":"2022","unstructured":"Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the phase III SPR1NT trial. Nat Med. 2022;28(7):1390\u20137. https:\/\/doi.org\/10.1038\/s41591-022-01867-3","journal-title":"Nat Med"},{"key":"3715_CR42","doi-asserted-by":"publisher","unstructured":"Ruan Y, Zhuang N, Zheng XY. Signal mining of hemorrhage events related to Ticagrelor and clopidogrel based on Faers database. Chin Pharm. 2023;32(4):102\u20136. https:\/\/doi.org\/10.3969\/j.issn.1006-4931.2023.04.024","DOI":"10.3969\/j.issn.1006-4931.2023.04.024"},{"issue":"6","key":"3715_CR43","doi-asserted-by":"publisher","first-page":"607","DOI":"10.1007\/s40264-020-00928-1","volume":"43","author":"KE Rees","year":"2020","unstructured":"Rees KE, Chyou TY, Nishtala PS. A disproportionality analysis of the adverse drug events associated with lurasidone in paediatric patients using the US FDA adverse event reporting system (FAERS). Drug Saf. 2020;43(6):607\u20139. https:\/\/doi.org\/10.1007\/s40264-020-00928-1","journal-title":"Drug Saf"},{"issue":"5","key":"3715_CR44","doi-asserted-by":"publisher","first-page":"626","DOI":"10.1177\/1945892420920505","volume":"34","author":"AE Wright","year":"2020","unstructured":"Wright AE, Davis ED, Khan M, Chaaban MR. Exploring balloon sinuplasty adverse events with the innovative OpenFDA database. Am J Rhinol Allergy. 2020;34(5):626\u201331. https:\/\/doi.org\/10.1177\/1945892420920505","journal-title":"Am J Rhinol Allergy"}],"container-title":["Orphanet Journal of Rare Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13023-025-03715-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s13023-025-03715-2\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13023-025-03715-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T12:55:48Z","timestamp":1746536148000},"score":1,"resource":{"primary":{"URL":"https:\/\/ojrd.biomedcentral.com\/articles\/10.1186\/s13023-025-03715-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,5,6]]},"references-count":44,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2025,12]]}},"alternative-id":["3715"],"URL":"https:\/\/doi.org\/10.1186\/s13023-025-03715-2","relation":{"references":[{"id-type":"doi","id":"10.1371\/journal.pone.0157753","asserted-by":"subject"}]},"ISSN":["1750-1172"],"issn-type":[{"value":"1750-1172","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,5,6]]},"assertion":[{"value":"15 October 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 April 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"6 May 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"This study complied with the principles of the Declaration of Helsinki. Because this was an observational study with the use of a global open database of anonymized information (openFDA), it did not involve therapeutic intervention or the collection of human samples and therefore did not require informed consent or ethics approval.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The authors declare no competing interests.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"215"}}