Nusinersen mitigates neuroinflammation in severe spinal muscular atrophy patients [PDF]
Communications Medicine, 2023 Nuzzo, Russo, Errico, D’Amico et al. investigate neuroinflammation in forty-eight pediatric spinal muscular atrophy patients before and after Nusinersen treatment.
Tommaso Nuzzo +13 more
doaj +5 more sources
Evaluation of inpatient and emergency department healthcare resource utilization and costs pre- and post-nusinersen for the treatment of spinal muscular atrophy using United States claims [PDF]
Journal of Comparative Effectiveness ResearchAim: Nusinersen, administered by intrathecal injection at a dose of 12 mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA).
Cong Zhu +6 more
doaj +3 more sources
Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy [PDF]
Biomolecules, 2022 Intrathecal delivery of Nusinersen–an antisense oligonucleotide that promotes survival motor neuron (SMN) protein induction–is an approved therapy for spinal muscular atrophy (SMA).
Francesco Errico +15 more
doaj +6 more sources
Experiences from treating seven adult 5q spinal muscular atrophy patients with Nusinersen [PDF]
Therapeutic Advances in Neurological Disorders, 2020 Background: The antisense oligonucleotide Nusinersen recently became the first approved drug against spinal muscular atrophy (SMA). It was approved for all ages, albeit the clinical trials were conducted exclusively on children.
Elisabeth Jochmann +11 more
doaj +2 more sources
Treatment expectations and perception of therapy in adult patients with spinal muscular atrophy receiving nusinersen [PDF]
European Journal of Neurology, 2021 Background and purpose: This was an investigation of treatment expectations and of the perception of therapy in adult patients with 5q-associated spinal muscular atrophy (5q-SMA) receiving nusinersen.
René Günther +2 more
exaly +3 more sources
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy [PDF]
Orphanet Journal of Rare Diseases, 2021 Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse.
Maren Freigang +12 more
doaj +2 more sources
Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy [PDF]
Orphanet Journal of Rare DiseasesBackground Spinal muscular atrophy (SMA) is a rare neurodegenerative disease that significantly affects multiple systems in children. Nusinersen, the first approved treatment for SMA, enhances SMN protein production by targeting the RNA splicing site of ...
Wenjing Li +3 more
doaj +2 more sources
Annals of Indian Academy of Neurology, 2020 Background: Nusinersen has recently been approved and more widely used as first-line treatment of spinal muscular atrophy (SMA). This study aimed to evaluate the real-world experience of nusinersen use for patients with a broad spectrum of SMA.
Ae Ryoung Kim +7 more
doaj +2 more sources
Full-Length SMN Transcript in Extracellular Vesicles as Biomarker in Individuals with Spinal Muscular Atrophy Type 2 Treated with Nusinersen [PDF]
, 2023 Three therapeutic strategies have radically changed the therapeutic scenario for spinal muscular atrophy (SMA). However, therapeutic response differs between individuals.
Selena Trifunov +14 more
openalex +4 more sources
Nusinersen treatment and cerebrospinal fluid neurofilaments: An explorative study on Spinal Muscular Atrophy type 3 patients [PDF]
Journal of Cellular and Molecular Medicine, 2020 The antisense oligonucleotide Nusinersen has been recently licensed to treat spinal muscular atrophy (SMA). Since SMA type 3 is characterized by variable phenotype and milder progression, biomarkers of early treatment response are urgently needed.
Irene Faravelli +2 more
exaly +3 more sources