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Effect Of Nusinersen On Motor, Respiratory And Bulbar Function

Di: Ava

Publications Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy. 13 February, 2023 The disease-modifying therapies (DMTs) (nusinersen, onasemnogene abeparvovec, and risdiplam) have improved survival, motor function, and functional muscle strength in patients with SMA, but their effects on lung function and respiratory muscle strength need further clarification. Effect of Nusinersen on Respiratory and Bulbar Function in Children with Spinal Muscular Atrophy: Real-World Experience from a Single Center

In the present study, respiratory function data from the control group were compared with those obtained in a new cohort of 27 infants and children with SMA1 who were treated with nusinersen. For the very first time, we were able to determine whether and how nusinersen affects respiratory function in the most severe form of the disease.

motor neuron disease | PPT

Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy Nusinersen: A Review in 5q Spinal Muscular Atrophy Nusinersen mitigates neuroinflammation in severe spinal muscular atrophy patients Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy

Prof. Dr. med. Maja von der Hagen

Conclusions: Nusinersen appears to enhance respiratory function and improve motor outcomes in SMA patients, especially with early treatment. However, further research is needed to fully understand its mechanisms and long-term effects on respiratory health in SMA.

This study seeks to assess the impact of nusinersen, when combined with standard care, on bulbar function, respiratory function, and the necessity for respiratory support among pediatric patients with spinal muscular atrophy (SMA). Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function.中文翻译： nusinersen 对早发性脊髓性肌萎缩症运动、呼吸和延髓功能的影响。 Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function.

Our findings confirm the increasing real-world evidence that treatment with nusinersen has a dramatic influence on disease progression in patients with SMA type 1 – especially regarding motor function and survival. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor Abstract It is unclear how improvements in peripheral motor function in children with spinal muscular atrophy (SMA), treated with nusinersen, translate into clinically significant respiratory/sleep outcomes. The disease-modifying therapies (DMTs) (nusinersen, onasemnogene abeparvovec, and risdiplam) have improved survival, motor function, and functional muscle strength in patients with SMA, but their effects on lung function and respiratory muscle strength need further clarification.

Effect of Nusinersen on Respiratory and Bulbar Function in Children with Spinal Muscular Atrophy: CorrespondenceNeuropediatrics. 2024 Oct 22. doi: 10.1055/a-2434-6190. Online ahead of print.

Publikationen von Steiner, Elisabeth

Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function.

In contrast to clinical trial data with restricted patient populations and short observation periods, we here report real-world evidence on a broad spectrum of patients with early-onset SMA treated with nusinersen focusing on effects regarding motor milestones, and respiratory and bulbar insufficiency during the first years of treatment. Respiratory function improvements were frequently observed, particularly in younger patients and those treated earlier. Conclusions: Nusinersen appears to enhance respiratory function and improve motor outcomes in SMA patients, especially with early treatment. Our study suggested that 14 months of treatment with nusinersen was effective and improved the motor function of children with SMA types 1, 2, or 3. In addition, disease duration and age at treatment initiation were negatively correlated with treatment outcome in the patients.

Spinal muscular atrophy (SMA) is a genetic progressive neuromuscular disease. Nusinersen is the first disease modifying drug approved to treat patients with SMA. Our study aimed to evaluate the efficacy of nusinersen treatment on motor function in Lung function was collected retrospectively for up to 2 years prior to nusinersen initiation. Change in lung function was assessed using mixed effects linear regression models, while PSG and muscle function were compared using the Wilcoxon signed-rank test. Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function.

Pechmann, Astrid; Behrens, Max; Dörnbrack, Katharina; Tassoni, Adrian; Stein, Sabine; Vogt, Sibylle; Zöller, Daniela; Bernert, Günther; Hagenacker, Tim; Schara-Schmidt, Ulrike; Schwersenz, Inge; Walter, Maggie C.; Baumann, Matthias; Baumgartner, Manuela; Deschauer, Marcus; Eisenkölbl, Astrid; Flotats-Bastardas, Marina; Hahn, Andreas; Horber Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy Article Full-text available Jul 2022

Inferential analysis evaluates the effect of age at diagnosis, age at start of treatment, SMN2 copy number (2 SMN2 copies versus ≥3 SMN2 copies), gender, baseline CHOP INTEND score and past time from baseline on changes in CHOP INTEND score. d.f. = degree of freedom. Publications Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy. 13 February, 2023

Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function. The drug nusinersen is applied to improve motor function in patients with spinal muscle atrophy (SMA). However, research on the effects of this treatment on lung function is lacking.

It is unclear how improvements in peripheral motor function in children with spinal muscular atrophy (SMA), treated with nusinersen, translate into clinically significant respiratory/sleep outcomes. In contrast to clinical trial data with restricted patient populations and short observation periods, we report here real-world evidence on a broad spectrum of patients with early-onset spinal muscular atrophy treated with nusinersen focusing on effects regarding motor milestones, and respiratory and bulbar insufficiency during the first years Abstract Background: We report the 4-year follow-up in type I patients treated with nusinersen and the changes in motor, respiratory and bulbar function in relation to subtype, age and SMN2 copy number. Methods: The study included SMA 1 patients with at least one assessment after 12, 24 and 48 months from the first dose of nusinersen.

Abstract Background We report the 4-year follow-up in type I patients treated with nusinersen and the changes in motor, respiratory and bulbar function in relation to subtype, age and SMN2 copy num Major improvements in motor function are seen in children younger than 2 years at the start of treatment. Bulbar and respiratory function needs to be closely monitored, as these functions do not improve equivalent to motor function.

Pechmann A, Behrens M, Dörnbrack K, Tassoni A, Stein S, Vogt S, et al. SMArtCARE study group Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy.