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.
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
Description
ISSN:1872-7131
DOI:10.1016/j.braindev.2026.104520