BACKGROUNDSpinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein. METHODSWe conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety trial of nusinersen in infants with spinal muscular atrophy. The primary end points were a motor-milestone response (defined according to results on the Hammersmith Infant Neurological Examination) and event-free survival (time to death or the use of permanent assisted ventilation). Secondary end points included overall survival and subgroup analyses of event-free survival according to disease duration at screening. Only the first primary end point was tested in a prespecified interim analysis. To control the overall type I error rate at 0.05, a hierarchical testing strategy was used for the second primary end point and the secondary end points in the final analysis. RESULTSIn the interim analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (21 of 51 infants [41%] vs. 0 of 27 [0%], P<0.001), and this result prompted early termination of the trial. In the final analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (37 of 73 infants [51%] vs. 0 of 37 [0%]), and the likelihood of event-free survival was higher in the nusinersen group than in the control group (hazard ratio for death or the use of permanent assisted ventilation, 0.53; P = 0.005). The likelihood of overall survival was higher in the nusinersen group than in the control group (hazard ratio for death, 0.37; P = 0.004), and infants with a shorter disease duration at screening were more likely than those with a longer disease duration to benefit from nusinersen. The incidence and severity of adverse events were similar in the two groups. CONCLUSIONSAmong infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug. (Funded by Biogen and Ionis Pharmaceuticals; ENDEAR ClinicalTrials.gov number, NCT02193074.)
Shoulder subluxation in hemiplegic patients has been recognized as a difficult problem to manage. In the study contained herein, our aims are to evaluate shoulder subluxation, to clarify if shoulder subluxation causes pain, and to discuss the treatment of shoulder subluxation. The study included 75 hemiplegic patients with shoulder subluxation. Each patient was evaluated for the degree of shoulder pain, motor recovery of the upper limb, and shoulder range of motion. Some indexes for evaluating subluxation were measured with radiographs of the shoulders. Arthrograms of the affected shoulder joint were taken in 23 patients. The following results were found: (1) shoulder pain was significant more frequently in left hemiplegia; (2) vertical disparity was strongly correlated with discrepancy of the descendant ratio; (3) severe inferior subluxation had a tendency to show medial displacement of the humeral head; (4) there were correlations between shoulder pain and shoulder range of motion, especially external rotation; (5) adhesive changes in the arthrograms were seen in most subjects. These results indicate that there is no relation between shoulder subluxation and pain, and adhesive capsulitis is a main cause of shoulder pain. We conclude that correct positioning and shoulder range of motion exercises are advisable in hemiplegic patients with shoulder subluxation.
These results do not support a strong relationship between shoulder subluxation and pain after stroke. Appropriate precautions should be taken to prevent range of motion limitations that may result in shoulder pain.
Ikai T, Kamikubo T, Takehara I, Nishi M, Miyano M: Dynamic postural control in patients with hemiparesis. Am J Phys Med Rehabil 2003;82:463-469.Objective: Decreased postural stability is a common problem associated with hemiparesis secondary to stroke. The purpose of this study was to evaluate dynamic postural control in patients with hemiparesis and in normal subjects matched for age.Design: Quantitative posturography (EquiTest System) was performed to assess the response of subjects to sudden perturbations. A total of 59 patients with hemiparesis and 98 healthy volunteers were evaluated. All the patients were able to walk inside their house without lower limb orthoses. Both the patients and the healthy volunteers were subjected to forward and backward perturbations while standing on a movable force platform. Balance responses were analyzed in terms of weight symmetry, latency, amplitude (relative response strength), and strength symmetry. They were also subjected to toes-up and toesdown perturbations to evaluate their response to a disruptive balance force. Results:The response latency to perturbations was longer and the response strength was weaker on the paretic side of patients with hemiparesis. The dynamic postural control was impaired in patients with hemiparesis as compared with healthy subjects. Conclusion:The results suggest that patients with hemiparesis tend to fall easily and that the risk of falls toward the paretic side is high.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.