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Results from multiple trials of SMN1gene-replacement therapy (Zolgensma; Avexis, Bannockburn, IL) show promise for single-dose treatment of spinal muscular atrophy (SMA). In the STRIVE phase 3 study for treatment of spinal muscular atrophy, 22 infants under age 6 months, diagnosed genetically (biallelic SMN1 mutations/deletions, 2 SMN2 copies), were treated. Of the 15 babies treated more than 13.6 months or who discontinued the study before then, 13 (87%) survived without needing permanent ventilation, in contrast to the 25-50% survival rate for children with SMA when untreated....
Treatment with inebilizumab (VielaBio, Gaithersburg, MD) reduced relapses of neuromyelitis optica spectrum disorder (NMOSD) by 77% after 28 weeks of treatment. Response to treatment with inebilizumab for NMOSD was safe and effective such that the trial was terminated early as it was deemed unethical not to treat participants in the placebo arm of a phase 3 trial (NCT02200770). After this judgement was made, all participants in the placebo arm began receiving inebilizumab on an open-label extension basis. Treatment with inebilizumab also reduced worsening from baseline score on...
Using a wearable technology (PKG) (Personal Kinetograph; Global Kinetics, Portsmouth, NH) cleared by the Food and Drug Administration (FDA), over 40,000 reports from individuals with Parkinson’s disease (PD) have been recorded and used by more than 200 Parkinson’s care centers. The PKG provides objective ambulatory measures of PD symptoms, including bradykinesia, dyskinesia, and tremor. Analysis of baseline data from the PKG showed that 54% of people reporting clinical measures with PKG had uncontrolled but likely treatable bradykinesia and 10% had uncontrolled, but likely...
Emmanuelle A. D. Schindler, MD, PhD; and Christopher Gottschalk, MD
Gordon H. Baltuch, MD, PhD
Saif A. Bushnaq, MD; and Sunil A. Sheth, MD