Results 11 to 20 of about 7,965 (282)
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 +7 more sources
Scientific rationale for a higher dose of nusinersen
Annals of Clinical and Translational Neurology, 2022 Objective The long‐term favorable safety profile of nusinersen provides an opportunity to consider a higher dose. We report on the relationships between nusinersen cerebrospinal fluid (CSF) exposure, biomarker levels, and clinical efficacy.
Richard S. Finkel +10 more
doaj +4 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
Development of gene therapies—lessons from nusinersen [PDF]
Gene Therapy, 2017 The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the Food and Drug Administration ...
Li Xu +3 more
openalex +4 more sources
Neurology InternationalBackground: Spinal muscular atrophy (SMA) is an inherited neuromuscular disease characterized by progressive muscle weakness and atrophy due to the absence of the survival motor neuron 1 (SMN1) gene.
Anna Lemska +4 more
doaj +3 more sources
Nusinersen Treatment in Adults With Spinal Muscular Atrophy [PDF]
Neurology Clinical Practice, 2021 To determine changes in motor and respiratory function after treatment with nusinersen in adults with spinal muscular atrophy (SMA) during the first two years of commercial availability in the USA.Data were collected prospectively on adult (age >17 years at treatment initiation) SMA participants in the Pediatric Neuromuscular Clinical Research (PNCR ...
Tina Duong +17 more
openalex +4 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
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
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
Neurology InternationalSpinal 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 ...
Asma AlTawari +12 more
doaj +2 more sources