• 48-week phase 3 RCT evaluating efficacy and safety of drisapersen 6 mg/kg/week• The pre-specified analyses did not meet statistical significance• Due to increased data variation, statistical power for 6MWD was reduced from to 53%• Evidence for a greater treatment benefit was seen in a selected population post-hoc• Most common adverse events were injection-site reactions and subclinical proteinuria
Falls are a major determinant of poor quality of life, immobilization, and reduced life expectancy in people affected by Parkinson’s disease (PD) and in older adults more generally. Although many questions remain, recent research has advanced the understanding of this complex problem.
Kv7 channels mediate the voltage-gated M-type potassium current. Reduction of M current due to KCNQ2 mutations causes early onset epileptic encephalopathies (EOEEs). Mutations in STXBP1 encoding the syntaxin binding protein 1 can produce a phenotype similar to that of KCNQ2 mutations, suggesting a possible link between STXBP1 and Kv7 channels.