{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,10]],"date-time":"2026-02-10T01:33:02Z","timestamp":1770687182290,"version":"3.49.0"},"reference-count":46,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T00:00:00Z","timestamp":1717459200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100005614","name":"Biogen Inc","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100005614","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["www.mdpi.com"],"crossmark-restriction":true},"short-container-title":["Neurology International"],"abstract":"<jats:p>Spinal muscular atrophy is a neuromuscular genetic condition associated with progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide therapy approved for the treatment of 5q spinal muscular atrophy in pediatric and adult patients. The objective of this clinical case series is to describe the efficacy and safety of nusinersen in treating spinal muscular atrophy in 20 pediatric and 18 adult patients across six treatment centers in Kuwait. Functional motor assessments (Children\u2019s Hospital of Philadelphia Infant Test of Neuromuscular Disorders, Hammersmith Functional Motor Scale Expanded, and Revised Upper Limb Module) were used to assess changes in motor function following nusinersen treatment. The safety assessment involved clinical monitoring of adverse events. The results demonstrate clinically meaningful or considerable improvement in motor performance for nearly all patients, lasting over 4 years in some cases. A total of 70% of patients in the pediatric cohort and 72% of patients in the adult cohort achieved a clinically meaningful improvement in motor function following nusinersen treatment. Additionally, nusinersen was well-tolerated in both cohorts. These findings add to the growing body of evidence relating to the clinical efficacy and safety of nusinersen.<\/jats:p>","DOI":"10.3390\/neurolint16030047","type":"journal-article","created":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T05:17:30Z","timestamp":1717478250000},"page":"631-642","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait"],"prefix":"10.3390","volume":"16","author":[{"given":"Asma","family":"AlTawari","sequence":"first","affiliation":[{"name":"Pediatric Department, Neurology Unit, Al Sabah Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mohammad","family":"Zakaria","sequence":"additional","affiliation":[{"name":"Pediatrics Department, Al Adan Hospital, Hadiya 47000, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3363-0370","authenticated-orcid":false,"given":"Walaa","family":"Kamel","sequence":"additional","affiliation":[{"name":"Neurology Department, Ibn Sina Hospital, Shuwaikh Industrial 70050, Kuwait"},{"name":"Neurology Department, Faculty of Medicine, Beni-Suef University, Beni-Suef 62521, Egypt"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nayera","family":"Shaalan","sequence":"additional","affiliation":[{"name":"Neurology Department, Ibn Sina Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gamal","family":"Elghazawi","sequence":"additional","affiliation":[{"name":"Pediatric Department, Neurology Unit, Al Sabah Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mohamed","family":"Ali","sequence":"additional","affiliation":[{"name":"Pediatric Department, Neurology Unit, Al Sabah Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dalia","family":"Salota","sequence":"additional","affiliation":[{"name":"Pediatric Department, Neurology Unit, Al Sabah Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amr","family":"Attia","sequence":"additional","affiliation":[{"name":"Pediatric Department, Neurology Unit, Al Sabah Hospital, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ehab","family":"Elanany","sequence":"additional","affiliation":[{"name":"Pediatrics Department, Al Adan Hospital, Hadiya 47000, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Osama","family":"Shalaby","sequence":"additional","affiliation":[{"name":"Pediatric Department, Al Jahra Hospital, Al Jahra 003200, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fatema","family":"Alqallaf","sequence":"additional","affiliation":[{"name":"Pediatric Department, Neurology Unit, Mubarak Hospital, Jabriya 46300, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vesna","family":"Mitic","sequence":"additional","affiliation":[{"name":"Pediatric Department, Al Farwaniya Hospital, Al Farwaniya 85000, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laila","family":"Bastaki","sequence":"additional","affiliation":[{"name":"Kuwait Medical Genetics Center, Shuwaikh Industrial 70050, Kuwait"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2024,6,4]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","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":"ref_2","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1186\/s13023-017-0671-8","article-title":"Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy\u2014A literature review","volume":"12","author":"Verhaart","year":"2017","journal-title":"Orphanet J. 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