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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....
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...
Post hoc analysis shows that participants who took 100 mg or 200 mg of lasmitidan had statistically significant increases in the rate of pain relief and freedom from the most bothersome symptom (MBS) (ie, nausea, photophobia, or phonophobia) (P < .001 starting as early as 30 minutes after administration. Participants who rated themselves as nonresponders, poor responders, or good responder to triptans did not have significant differences in their responses to 200 mg lasmiditan in any measure (freedom from pain at 2 hours, freedom from MBS, or pain relief). Participants who...
Bob Azelby, MBA
Deena Kuruvilla, MD
Niushen Zhang, MD