{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T02:51:49Z","timestamp":1791341509561,"version":"4.3.1"},"reference-count":83,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2025,1,7]],"date-time":"2025-01-07T00:00:00Z","timestamp":1736208000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Pharmacol."],"abstract":"<jats:p>Onasemnogene Abeparvovec (Zolgensma) is a gene therapy for the treatment of Spinal Muscular Atrophy (SMA) with improved motor neuron function and the potential for a singular treatment. Information on its adverse drug reactions is mainly from clinical trials and real-world studies with extensive sample sizes are lacking. In this study, we analyzed the U.S. Food and Drug Administration\u2019s Adverse Event Reporting System (FAERS) database to assess the drug safety profile of Zolgensma. A total of 1951 adverse event reports associated with onasemnogene abeparvovec (Zolgensma), containing 778 import important medical event (IME) signals, were identified from the FAERS database, and multiple disproportionate analysis algorithms were used to determine the significance of these adverse events. This study identified 281 onasemnogene abeparvovec-related adverse events (AEs), including some significant adverse events not mentioned in the product labelling. Elevated liver enzymes, fever, vomiting, and thrombocytopenia were the most common adverse reactions. Most adverse events manifested within the initial month of onasemnogene abeparvovec use, especially the first 8\u00a0days, but some may still occur after 1\u00a0year of treatment. Sex-specific scrutiny revealed differing risk levels for adverse events among women and men. Thrombocytopenia and thrombotic microangiopathy are more common in patients weighing \u22658.5\u00a0kg, and changes in renal function need to be closely monitored if thrombotic microangiopathy occurs. The above findings provide valuable insights into optimizing the utilization of onasemnogene abeparvovec, improving its effectiveness, and minimizing potential side effects, thereby greatly facilitating its practical application in clinical settings.<\/jats:p>","DOI":"10.3389\/fphar.2024.1475884","type":"journal-article","created":{"date-parts":[[2025,1,7]],"date-time":"2025-01-07T07:26:24Z","timestamp":1736234784000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":9,"title":["Comprehensive analysis of adverse events associated with onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy patients: insights from FAERS database"],"prefix":"10.3389","volume":"15","author":[{"given":"Wenwen","family":"Zhang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yizhen","family":"Yin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mengyuan","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caixia","family":"Ye","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruiling","family":"Yan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruiman","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2025,1,7]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1002\/mus.24497","article-title":"Spinal muscular atrophy: diagnosis and management in a new therapeutic era","volume":"51","author":"Arnold","year":"2015","journal-title":"Muscle Nerve"},{"key":"B2","doi-asserted-by":"publisher","first-page":"162","DOI":"10.3889\/oamjms.2019.010","article-title":"The diagnostic and clinical approach to pediatric myocarditis: a review of the current literature","volume":"7","author":"Bejiqi","year":"2019","journal-title":"Open Access Maced. J. Med. Sci."},{"key":"B3","article-title":"Onasemnogene abeparvovec","volume-title":"LiverTox: clinical and research information on drug-induced liver injury","year":"2012"},{"key":"B4","doi-asserted-by":"publisher","first-page":"592","DOI":"10.1038\/s41434-022-00341-6","article-title":"Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study","volume":"30","author":"Bitetti","year":"2023","journal-title":"Gene Ther."},{"key":"B5","doi-asserted-by":"publisher","first-page":"995","DOI":"10.1007\/s40263-022-00941-1","article-title":"Onasemnogene abeparvovec: a review in spinal muscular atrophy","volume":"36","author":"Blair","year":"2022","journal-title":"CNS Drugs"},{"key":"B6","doi-asserted-by":"publisher","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","article-title":"Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy","volume":"74","author":"Chand","year":"","journal-title":"J. Hepatol."},{"key":"B7","doi-asserted-by":"publisher","first-page":"685","DOI":"10.1038\/s41434-023-00401-5","article-title":"Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings","volume":"30","author":"Chand","year":"2023","journal-title":"Gene Ther."},{"key":"B8","doi-asserted-by":"publisher","first-page":"265","DOI":"10.1016\/j.jpeds.2020.11.054","article-title":"Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series","volume":"231","author":"Chand","year":"","journal-title":"J. Pediatr."},{"key":"B9","doi-asserted-by":"publisher","first-page":"3877","DOI":"10.1007\/s00018-018-2849-1","article-title":"The role of survival motor neuron protein (SMN) in protein homeostasis","volume":"75","author":"Chaytow","year":"2018","journal-title":"Cell. Mol. Life Sci. CMLS"},{"key":"B10","doi-asserted-by":"publisher","first-page":"945","DOI":"10.1038\/s41436-020-0754-0","article-title":"Spinal muscular atrophy diagnosis and carrier screening from genome sequencing data","volume":"22","author":"Chen","year":"2020","journal-title":"Genet. Med."},{"key":"B11","doi-asserted-by":"publisher","first-page":"269","DOI":"10.1002\/mus.27926","article-title":"Gene transfer therapy in children with spinal muscular atrophy: a single-center experience with a cohort of 25 children","volume":"68","author":"Chencheri","year":"2023","journal-title":"Muscle Nerve"},{"key":"B12","doi-asserted-by":"publisher","first-page":"543","DOI":"10.1007\/s40120-023-00444-1","article-title":"Risdiplam in patients previously treated with other therapies for spinal muscular atrophy: an interim analysis from the JEWELFISH study","volume":"12","author":"Chiriboga","year":"2023","journal-title":"Neurol. Ther."},{"key":"B13","doi-asserted-by":"publisher","first-page":"1235","DOI":"10.1016\/j.kint.2021.02.015","article-title":"Kidney disorders as serious adverse drug reactions of remdesivir in coronavirus disease 2019: a retrospective case-noncase study","volume":"99","author":"Chouchana","year":"2021","journal-title":"Kidney Int."},{"key":"B14","doi-asserted-by":"publisher","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging issues in AAV-mediated in vivo gene therapy","volume":"8","author":"Colella","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"B15","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1080\/14740338.2023.2219892","article-title":"An overview of methodological flaws of real-world studies investigating drug safety in the post-marketing setting","volume":"22","author":"Crisafulli","year":"2023","journal-title":"Expert Opin. Drug Saf."},{"key":"B16","doi-asserted-by":"publisher","first-page":"284","DOI":"10.1016\/S1474-4422(21)00001-6","article-title":"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","volume":"20","author":"Day","year":"","journal-title":"Lancet Neurol."},{"key":"B17","doi-asserted-by":"publisher","first-page":"632","DOI":"10.1186\/s12887-022-03671-x","article-title":"Advances and limitations for the treatment of spinal muscular atrophy","volume":"22","author":"Day","year":"2022","journal-title":"BMC Pediatr."},{"key":"B18","doi-asserted-by":"publisher","first-page":"1109","DOI":"10.1007\/s40264-021-01107-6","article-title":"Clinical trial and postmarketing safety of onasemnogene abeparvovec therapy","volume":"44","author":"Day","year":"","journal-title":"Drug Saf."},{"key":"B19","doi-asserted-by":"publisher","first-page":"489","DOI":"10.1007\/s40264-020-00913-8","article-title":"Sex differences in adverse drug reactions of metformin: a longitudinal survey study","volume":"43","author":"de Vries","year":"2020","journal-title":"Drug Saf."},{"key":"B20","doi-asserted-by":"publisher","first-page":"339","DOI":"10.1002\/acn3.51519","article-title":"Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy","volume":"9","author":"D\u2019Silva","year":"2022","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"B21","unstructured":"EudraVigilance system overview (EMA)\n          \n          \n          2016"},{"key":"B22","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.ejpn.2022.04.006","article-title":"Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: a systematic review of real-world study data","volume":"39","author":"Erdos","year":"2022","journal-title":"Eur. J. Paediatr. Neurol. EJPN Off. J. Eur. Paediatr. Neurol. Soc."},{"key":"B23","doi-asserted-by":"publisher","first-page":"39","DOI":"10.1177\/00185787231182562","article-title":"Onasemnogene abeparvovec: post-infusion efficacy and safety in patients with spinal muscular atrophy (SMA)\u2014a fondazione policlinico gemelli IRCCS experience","volume":"59","author":"Favia","year":"2024","journal-title":"Hosp. Pharm."},{"key":"B24","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1086\/338627","article-title":"Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy","volume":"70","author":"Feldk\u00f6tter","year":"2002","journal-title":"Am. J. Hum. Genet."},{"key":"B25","doi-asserted-by":"publisher","first-page":"389","DOI":"10.3233\/JND-221560","article-title":"Intrathecal onasemnogene abeparvovec for sitting, nonambulatory patients with spinal muscular atrophy: phase I ascending-dose study (STRONG)","volume":"10","author":"Finkel","year":"2023","journal-title":"J. Neuromuscul. Dis."},{"key":"B26","doi-asserted-by":"publisher","first-page":"810","DOI":"10.1212\/WNL.0000000000000741","article-title":"Observational study of spinal muscular atrophy type I and implications for clinical trials","volume":"83","author":"Finkel","year":"2014","journal-title":"Neurology"},{"key":"B27","doi-asserted-by":"publisher","first-page":"100817","DOI":"10.1016\/j.lanepe.2023.100817","article-title":"Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom","volume":"37","author":"Gowda","year":"2024","journal-title":"Lancet Reg. Health Eur."},{"key":"B28","doi-asserted-by":"publisher","first-page":"4266","DOI":"10.1182\/bloodadvances.2021006419","article-title":"Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy","volume":"6","author":"Guillou","year":"2022","journal-title":"Blood Adv."},{"key":"B29","doi-asserted-by":"publisher","first-page":"675897","DOI":"10.3389\/fimmu.2021.675897","article-title":"Challenges posed by immune responses to AAV vectors: addressing root causes","volume":"12","author":"Hamilton","year":"2021","journal-title":"Front. Immunol."},{"key":"B30","doi-asserted-by":"publisher","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"B31","doi-asserted-by":"publisher","first-page":"1865","DOI":"10.2147\/DDDT.S214174","article-title":"Disease modifying therapies for the management of children with spinal muscular atrophy (5q SMA): an update on the emerging evidence","volume":"16","author":"Hjartarson","year":"2022","journal-title":"Drug Des. devel. Ther."},{"key":"B32","doi-asserted-by":"publisher","first-page":"808","DOI":"10.1089\/hum.2020.167","article-title":"Adeno-associated virus-induced dorsal root ganglion pathology","volume":"31","author":"Hordeaux","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"B33","doi-asserted-by":"publisher","first-page":"1255","DOI":"10.1007\/s40265-019-01162-5","article-title":"Onasemnogene abeparvovec: first global approval","volume":"79","author":"Hoy","year":"2019","journal-title":"Drugs"},{"key":"B34","doi-asserted-by":"publisher","first-page":"2999","DOI":"10.1016\/j.ymthe.2023.07.020","article-title":"Liver injury in cynomolgus monkeys following intravenous and intrathecal scAAV9 gene therapy delivery","volume":"31","author":"Hudry","year":"2023","journal-title":"Mol. Ther. J. Am. Soc. Gene Ther."},{"key":"B35","doi-asserted-by":"publisher","first-page":"102","DOI":"10.1186\/cc11897","article-title":"Acute liver failure and elevated troponin-I: controversial results and significance?","volume":"17","author":"Jaber","year":"2013","journal-title":"Crit. Care Lond. Engl."},{"key":"B36","doi-asserted-by":"publisher","first-page":"1259293","DOI":"10.3389\/fped.2023.1259293","article-title":"High-sensitive cardiac troponin I (hs-cTnI) concentrations in newborns diagnosed with spinal muscular atrophy","volume":"11","author":"Johannsen","year":"2023","journal-title":"Front. Pediatr."},{"key":"B37","doi-asserted-by":"publisher","first-page":"235","DOI":"10.1007\/s40261-021-01006-9","article-title":"Correlations between SGLT-2 inhibitors and acute renal failure by signal detection using FAERS: stratified analysis for reporting country and concomitant drugs","volume":"41","author":"Katsuhara","year":"2021","journal-title":"Clin. Drug Investig."},{"key":"B38","doi-asserted-by":"publisher","first-page":"947","DOI":"10.1089\/hum.2016.160","article-title":"Adeno-associated virus gene therapy for liver disease","volume":"27","author":"Kattenhorn","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"B39","doi-asserted-by":"publisher","first-page":"413","DOI":"10.1002\/mus.27363","article-title":"Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy","volume":"64","author":"Kichula","year":"2021","journal-title":"Muscle Nerve"},{"key":"B40","doi-asserted-by":"publisher","first-page":"883","DOI":"10.1002\/ana.25101","article-title":"Natural history of infantile-onset spinal muscular atrophy","volume":"82","author":"Kolb","year":"2017","journal-title":"Ann. Neurol."},{"key":"B41","doi-asserted-by":"publisher","first-page":"726468","DOI":"10.3389\/fneur.2021.726468","article-title":"Recombinant adeno-associated virus serotype 9 gene therapy in spinal muscular atrophy","volume":"12","author":"Kotulska","year":"2021","journal-title":"Front. Neurol."},{"key":"B42","doi-asserted-by":"publisher","first-page":"287","DOI":"10.1016\/j.braindev.2021.12.006","article-title":"Short-term clinical outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy","volume":"44","author":"Lee","year":"2022","journal-title":"Brain Dev."},{"key":"B43","doi-asserted-by":"publisher","first-page":"1418","DOI":"10.1111\/jcpt.13472","article-title":"Thyroid dysfunction related to vascular endothelial growth factor receptor tyrosine kinase inhibitors: a real-world study based on FAERS","volume":"46","author":"Liao","year":"2021","journal-title":"J. Clin. Pharm. Ther."},{"key":"B44","doi-asserted-by":"publisher","first-page":"100300","DOI":"10.1016\/j.jhepr.2021.100300","article-title":"Novel vectors and approaches for gene therapy in liver diseases","volume":"3","author":"Maestro","year":"2021","journal-title":"JHEP Rep."},{"key":"B45","doi-asserted-by":"publisher","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat. Med."},{"key":"B46","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.pediatrneurol.2021.01.012","article-title":"Clinical experience with gene therapy in older patients with spinal muscular atrophy","volume":"118","author":"Matesanz","year":"2021","journal-title":"Pediatr. Neurol."},{"key":"B47","doi-asserted-by":"publisher","first-page":"1075","DOI":"10.1080\/14712598.2022.2066471","article-title":"Onasemnogene abeparvovec for the treatment of spinal muscular atrophy","volume":"22","author":"McMillan","year":"2022","journal-title":"Expert Opin. Biol. Ther."},{"key":"B48","doi-asserted-by":"publisher","first-page":"834","DOI":"10.1001\/jamaneurol.2021.1272","article-title":"Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy","volume":"78","author":"Mendell","year":"2021","journal-title":"JAMA Neurol."},{"key":"B49","doi-asserted-by":"publisher","first-page":"391","DOI":"10.1038\/s41434-024-00456-y","article-title":"Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort","volume":"31","author":"Mendon\u00e7a","year":"2024","journal-title":"Gene Ther."},{"key":"B50","doi-asserted-by":"publisher","first-page":"832","DOI":"10.1016\/S1474-4422(21)00251-9","article-title":"Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial","volume":"20","author":"Mercuri","year":"2021","journal-title":"Lancet Neurol."},{"key":"B51","doi-asserted-by":"publisher","first-page":"1230889","DOI":"10.3389\/fneur.2023.1230889","article-title":"Case report: a case of spinal muscular atrophy in a preterm infant: risks and benefits of treatment","volume":"14","author":"Nigro","year":"2023","journal-title":"Front. Neurol."},{"key":"B52","doi-asserted-by":"publisher","first-page":"11939","DOI":"10.3390\/ijms241511939","article-title":"Spinal muscular atrophy: the past, present, and future of diagnosis and treatment","volume":"24","author":"Nishio","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"B53","unstructured":"ZOLGENSMA package insert\n          \n          \n          2024"},{"key":"B54","doi-asserted-by":"publisher","first-page":"140","DOI":"10.18773\/austprescr.2022.044","article-title":"Onasemnogene abeparvovec for spinal muscular atrophy","volume":"45","year":"2022","journal-title":"Aust. Prescr."},{"key":"B55","doi-asserted-by":"publisher","first-page":"e36197","DOI":"10.7759\/cureus.36197","article-title":"Gene therapy for spinal muscular atrophy (SMA): a review of current challenges and safety considerations for onasemnogene abeparvovec (Zolgensma)","volume":"15","author":"Ogbonmide","year":"2023","journal-title":"Cureus"},{"key":"B56","doi-asserted-by":"publisher","first-page":"1489","DOI":"10.1002\/hep.21640","article-title":"Elevated troponin I levels in acute liver failure: is myocardial injury an integral part of acute liver failure?","volume":"45","author":"Parekh","year":"2007","journal-title":"Hepatol. Balt. Md"},{"key":"B57","doi-asserted-by":"publisher","first-page":"136","DOI":"10.1007\/s00701-024-06028-8","article-title":"Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS","volume":"166","author":"Perera","year":"2024","journal-title":"Acta Neurochir."},{"key":"B58","article-title":"Spinal muscular atrophy","volume-title":"GeneReviews\u00ae","author":"Prior","year":"2020"},{"key":"B59","doi-asserted-by":"publisher","first-page":"1404","DOI":"10.1002\/jcsm.13486","article-title":"Improved therapeutic approach for spinal muscular atrophy via ubiquitination-resistant survival motor neuron variant","volume":"15","author":"Rhee","year":"2024","journal-title":"J. Cachexia Sarcopenia Muscle"},{"key":"B60","doi-asserted-by":"publisher","first-page":"5104","DOI":"10.3390\/jcm11175104","article-title":"Did the COVID-19 pandemic affect contrast media-induced adverse drug reaction\u2019s reporting? A pharmacovigilance study in southern Italy","volume":"11","author":"Rossi","year":"2022","journal-title":"J. Clin. Med."},{"key":"B61","doi-asserted-by":"publisher","first-page":"394","DOI":"10.3390\/ph17030394","article-title":"Real-world safety data of the orphan drug onasemnogene abeparvovec (Zolgensma\u00ae) for the SMA rare disease: a pharmacovigilance study based on the EMA adverse event reporting system","volume":"17","author":"Ruggiero","year":"2024","journal-title":"Pharm. Basel Switz."},{"key":"B62","doi-asserted-by":"publisher","first-page":"1931","DOI":"10.1016\/j.clinthera.2018.09.015","article-title":"On the policy of the Italian government in the discovery, development, and access to Medicines","volume":"40","author":"Scavone","year":"2018","journal-title":"Clin. Ther."},{"key":"B63","doi-asserted-by":"publisher","first-page":"1","DOI":"10.3233\/JND-190424","article-title":"Advances in treatment of spinal muscular atrophy - new phenotypes, new challenges, new implications for care","volume":"7","author":"Schorling","year":"2020","journal-title":"J. Neuromuscul. Dis."},{"key":"B64","doi-asserted-by":"publisher","first-page":"425","DOI":"10.3233\/JND-230122","article-title":"Real-world outcomes in patients with spinal muscular atrophy treated with onasemnogene abeparvovec monotherapy: findings from the RESTORE registry","volume":"11","author":"Servais","year":"2024","journal-title":"J. Neuromuscul. Dis."},{"key":"B65","doi-asserted-by":"publisher","first-page":"862508","DOI":"10.3389\/fphar.2022.862508","article-title":"Post-marketing safety concerns with secukinumab: a disproportionality analysis of the FDA adverse event reporting system","volume":"13","author":"Shu","year":"","journal-title":"Front. Pharmacol."},{"key":"B66","doi-asserted-by":"publisher","first-page":"789","DOI":"10.2147\/CLEP.S365513","article-title":"A real-world disproportionality analysis of olaparib: data mining of the public version of FDA adverse event reporting system","volume":"14","author":"Shu","year":"","journal-title":"Clin. Epidemiol."},{"key":"B67","doi-asserted-by":"publisher","first-page":"88","DOI":"10.1186\/s12883-023-03133-6","article-title":"Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study","volume":"23","author":"Stettner","year":"2023","journal-title":"BMC Neurol."},{"key":"B68","doi-asserted-by":"publisher","first-page":"1001","DOI":"10.1177\/1060028020914274","article-title":"Onasemnogene abeparvovec-xioi: gene therapy for spinal muscular atrophy","volume":"54","author":"Stevens","year":"2020","journal-title":"Ann. Pharmacother."},{"key":"B69","doi-asserted-by":"publisher","first-page":"1381","DOI":"10.1038\/s41591-022-01866-4","article-title":"Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial","volume":"28","author":"Strauss","year":"","journal-title":"Nat. Med."},{"key":"B70","doi-asserted-by":"publisher","first-page":"1390","DOI":"10.1038\/s41591-022-01867-3","article-title":"Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial","volume":"28","author":"Strauss","year":"","journal-title":"Nat. Med."},{"key":"B71","article-title":"Actualizaci\u00f3n en tratamientos de La atrofia muscular espinal","author":"Torricelli","year":"2022"},{"key":"B72","doi-asserted-by":"publisher","first-page":"740","DOI":"10.1089\/hum.2021.255","article-title":"Single-dose intrathecal dorsal root ganglia toxicity of onasemnogene abeparvovec in cynomolgus monkeys","volume":"33","author":"Tukov","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"B73","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1016\/S2352-4642(21)00287-X","article-title":"Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study","volume":"6","author":"Wei\u00df","year":"2022","journal-title":"Lancet Child. Adolesc. Health"},{"key":"B74","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1055\/a-1538-4936","article-title":"Thrombotic microangiopathy (TMA) after gene replacemant therapy (GRT) due to spinal muscular atrophy: case summary and recommendations for treatment","volume":"234","author":"Witte","year":"2022","journal-title":"Klin. Padiatr."},{"key":"B75","doi-asserted-by":"publisher","first-page":"1129730","DOI":"10.3389\/fphar.2023.1129730","article-title":"Pneumocystis jirovecii pneumonia associated with immune checkpoint inhibitors: a systematic literature review of published case reports and disproportionality analysis based on the FAERS database","volume":"14","author":"Xia","year":"2023","journal-title":"Front. Pharmacol."},{"key":"B76","doi-asserted-by":"publisher","first-page":"945","DOI":"10.1038\/s44321-024-00037-x","article-title":"Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor","volume":"16","author":"Xie","year":"2024","journal-title":"EMBO Mol. Med."},{"key":"B77","doi-asserted-by":"publisher","first-page":"431","DOI":"10.1111\/jpc.16340","article-title":"Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: a systematic review and meta-analysis","volume":"59","author":"Yang","year":"2023","journal-title":"J. Paediatr. Child. Health"},{"key":"B78","doi-asserted-by":"publisher","first-page":"772768","DOI":"10.3389\/fphar.2021.772768","article-title":"Adverse events during pregnancy associated with entecavir and adefovir: new insights from a real-world analysis of cases reported to FDA adverse event reporting system","volume":"12","author":"Yang","year":"2021","journal-title":"Front. Pharmacol."},{"key":"B79","doi-asserted-by":"publisher","first-page":"808","DOI":"10.1212\/WNL.0000000000200676","article-title":"Child neurology: pathologically confirmed thrombotic microangiopathy caused by onasemnogene abeparvovec treatment for SMA","volume":"98","author":"Yazaki","year":"2022","journal-title":"Neurology"},{"key":"B80","doi-asserted-by":"publisher","first-page":"205","DOI":"10.1016\/S1474-4422(23)00419-2","article-title":"Challenges and opportunities in spinal muscular atrophy therapeutics","volume":"23","author":"Yeo","year":"2024","journal-title":"Lancet Neurol."},{"key":"B81","doi-asserted-by":"publisher","first-page":"19555","DOI":"10.1038\/s41598-022-23834-1","article-title":"A real-world pharmacovigilance study of FDA Adverse Event Reporting System (FAERS) events for osimertinib","volume":"12","author":"Yin","year":"2022","journal-title":"Sci. Rep."},{"key":"B82","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1007\/s40261-023-01320-4","article-title":"Safety concerns with nusinersen, risdiplam, and onasemnogene abeparvovec in spinal muscular atrophy: a real-world pharmacovigilance study","volume":"43","author":"Zhuang","year":"2023","journal-title":"Clin. Drug Investig."},{"key":"B83","doi-asserted-by":"publisher","first-page":"1338902","DOI":"10.3389\/fphar.2024.1338902","article-title":"Adverse drug events associated with linezolid administration: a real-world pharmacovigilance study from 2004 to 2023 using the FAERS database","volume":"15","author":"Zou","year":"2024","journal-title":"Front. Pharmacol."}],"container-title":["Frontiers in Pharmacology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fphar.2024.1475884\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,1,7]],"date-time":"2025-01-07T07:26:31Z","timestamp":1736234791000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fphar.2024.1475884\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1,7]]},"references-count":83,"alternative-id":["10.3389\/fphar.2024.1475884"],"URL":"https:\/\/doi.org\/10.3389\/fphar.2024.1475884","relation":{},"ISSN":["1663-9812"],"issn-type":[{"value":"1663-9812","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1,7]]},"article-number":"1475884"}}