Clinical Outcome of Adult Spinal Muscular Atrophy Patients Treated with Nusinersen: A Case Series Review

Lam, Keng; Wu, Albert T.

doi:10.7812/tpp/20.163

Cited by 2 publications

(6 citation statements)

References 9 publications

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“…Furthermore, a 2021 meta-analysis that included observational data from pediatric and adult patients with SMA types II and III also reported that nusinersen provided a favorable benefit in motor performance over a 10-14-month period [35]. In terms of safety, nusinersen was well-tolerated within both cohorts in this study, which is consistent with recently published observational studies [21,36,37]. In addition, the generally improved functional performance of patients treated with nusinersen is thought to be associated with enhanced function and/or survival of motor neurons caused by increased SMN protein production.…”

Section: Discussionsupporting

confidence: 88%

“…Nusinersen is an antisense oligonucleotide approved for the treatment 5q SMA in pediatric and adult patients [21]. It increases production of SMN protein in the central nervous system by altering splicing of SMN2 to promote exon 7 inclusion, thus improving motor neuron survival [22,23].…”

Section: Introductionmentioning

confidence: 99%

“…Studies have demonstrated that nusinersen improves motor function in SMA and increases survival rates in type I patients [7,22,24]. Nusinersen is also effective in treating symptoms of SMA in adults, and realworld data is increasingly providing an understanding of its potential benefits and tolerability in adult SMA [21,25].…”

Section: Introductionmentioning

confidence: 99%

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Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

Tawari,

Zakaria,

Kamel

et al. 2024

Preprint

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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’s 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. 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. 70% of patients in the pediatric cohort and 72% of patients in the adult cohort achieved 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.

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Section: Discussionsupporting

confidence: 88%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

Tawari,

Zakaria,

Kamel

et al. 2024

Preprint

View full text Add to dashboard Cite

show abstract

“…Nusinersen is an antisense oligonucleotide approved for the treatment 5q SMA in pediatric and adult patients [ 21 ]. It increases production of SMN protein in the central nervous system by altering the splicing of SMN2 to promote exon 7 inclusion, thus improving motor neuron survival [ 22 , 23 ].…”

Section: Introductionmentioning

confidence: 99%

“…Studies have demonstrated that nusinersen improves motor function in SMA and increases survival rates in type I patients [ 7 , 22 , 24 ]. Nusinersen is also effective in treating symptoms of SMA in adults, and real-world data are increasingly providing an understanding of its potential benefits and tolerability in adult SMA [ 21 , 25 ].…”

Section: Introductionmentioning

confidence: 99%

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

AlTawari,

Zakaria,

Kamel

et al. 2024

Neurology International

View full text Add to dashboard Cite

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’s 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.

show abstract

Clinical Outcome of Adult Spinal Muscular Atrophy Patients Treated with Nusinersen: A Case Series Review

Cited by 2 publications

References 9 publications

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

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