Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report.
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| Title: | Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report. |
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| Authors: | Okubo Y; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan. Electronic address: yokubom@yahoo.co.jp., Togashi N; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Morishita Y; Department of Rehabilitation and Developmental Support, Miyagi Children's Hospital, Sendai, Japan., Yoshida S; Department of Rehabilitation and Developmental Support, Miyagi Children's Hospital, Sendai, Japan., Kawaji T; Department of Physiological Examination, Miyagi Children's Hospital, Sendai, Japan., Kawashima A; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Katata Y; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Uneoka S; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Yagi Y; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Endo W; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Inui T; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan., Kato T; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Yokomura M; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Otsuki N; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Haginoya K; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan. Electronic address: khaginoya@kha.biglobe.ne.jp. |
| Source: | Brain & development [Brain Dev] 2026 Apr; Vol. 48 (2), pp. 104520. Date of Electronic Publication: 2026 Mar 06. |
| Publication Type: | Case Reports; Journal Article |
| Journal Info: | Publisher: Elsevier Country of Publication: Netherlands NLM ID: 7909235 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1872-7131 (Electronic) Linking ISSN: 03877604 NLM ISO Abbreviation: Brain Dev Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 41795276 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Okubo+Y%22">Okubo Y</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan. Electronic address: yokubom@yahoo.co.jp.<br /><searchLink fieldCode="AU" term="%22Togashi+N%22">Togashi N</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Morishita+Y%22">Morishita Y</searchLink>; Department of Rehabilitation and Developmental Support, Miyagi Children's Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Yoshida+S%22">Yoshida S</searchLink>; Department of Rehabilitation and Developmental Support, Miyagi Children's Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Kawaji+T%22">Kawaji T</searchLink>; Department of Physiological Examination, Miyagi Children's Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Kawashima+A%22">Kawashima A</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Katata+Y%22">Katata Y</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Uneoka+S%22">Uneoka S</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Yagi+Y%22">Yagi Y</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Endo+W%22">Endo W</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Inui+T%22">Inui T</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan.<br /><searchLink fieldCode="AU" term="%22Kato+T%22">Kato T</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Yokomura+M%22">Yokomura M</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Otsuki+N%22">Otsuki N</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Saito+K%22">Saito K</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Haginoya+K%22">Haginoya K</searchLink>; Department of Pediatric Neurology, Miyagi Children Hospital, Sendai, Japan. Electronic address: khaginoya@kha.biglobe.ne.jp. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%227909235%22">Brain & development</searchLink> [Brain Dev] 2026 Apr; Vol. 48 (2), pp. 104520. <i>Date of Electronic Publication: </i>2026 Mar 06. – Name: TypePub Label: Publication Type Group: TypPub Data: Case Reports; Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier%22">Elsevier </searchLink><i>Country of Publication: </i>Netherlands <i>NLM ID: </i>7909235 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1872-7131 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2203877604%22">03877604 </searchLink><i>NLM ISO Abbreviation: </i>Brain Dev <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41795276 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1016/j.braindev.2026.104520 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 104520 Titles: – TitleFull: Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Okubo Y – PersonEntity: Name: NameFull: Togashi N – PersonEntity: Name: NameFull: Morishita Y – PersonEntity: Name: NameFull: Yoshida S – PersonEntity: Name: NameFull: Kawaji T – PersonEntity: Name: NameFull: Kawashima A – PersonEntity: Name: NameFull: Katata Y – PersonEntity: Name: NameFull: Uneoka S – PersonEntity: Name: NameFull: Yagi Y – PersonEntity: Name: NameFull: Endo W – PersonEntity: Name: NameFull: Inui T – PersonEntity: Name: NameFull: Kato T – PersonEntity: Name: NameFull: Yokomura M – PersonEntity: Name: NameFull: Otsuki N – PersonEntity: Name: NameFull: Saito K – PersonEntity: Name: NameFull: Haginoya K IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 04 Text: 2026 Apr Type: published Y: 2026 Identifiers: – Type: issn-electronic Value: 1872-7131 Numbering: – Type: volume Value: 48 – Type: issue Value: 2 Titles: – TitleFull: Brain & development Type: main |
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