Optogenetic therapy appears safe in a small study of patients blind from advanced retinitis pigmentosa, with early efficacy signals described as promising. The findings also point to opportunities for technical refinement of the approach.
Nicotinamide riboside, 500 mg twice daily for 52 weeks, did not improve clinical outcomes in early Parkinson disease and was worse than placebo. In the phase 3 NOPARK trial of 393 patients, the MDS-UPDRS parts I-III score worsened by 2.72 points with nicotinamide riboside relative to placebo (P = .02), and nonmotor symptom burden worsened more on treatment (NMSS difference 4.22 points, P = .009). The findings do not support nicotinamide riboside as a disease-modifying treatment.
Why it matters: Phase 3 trial shows a proposed disease-modifying therapy for Parkinson disease was ineffective and worse than placebo.
Not routinely assessing gastric residual volume in ventilated critically ill children was noninferior to 6-hourly assessments for survival and ventilator-free days at 30 days (median 25 days in both groups; adjusted OR 0.95 [95% CI 0.86-1.05]). In the trial of 4700 children in pediatric intensive care units in the UK and Switzerland, dropping routine GRV checks also improved feeding: mean energy requirements achieved by 72 hours were 80.3% versus 76.8% (adjusted mean difference 3.2 percentage points, P < .001).
Why it matters: A large randomized noninferiority trial shows routine GRV checks can be dropped in PICU enteral feeding without harming outcomes.
Intravenous loberamisal, given once daily for 10 days on top of standard care, improved functional recovery after acute ischemic stroke in the phase 3 LAIS trial. Among 997 treated adults in China with NIHSS scores of 7 to 20 within 48 hours of onset, 69.7% reached a 90-day mRS score of 0 to 1 versus 56.3% with placebo (RR, 1.24), with no increase in adverse events, serious adverse events, or mortality.
Why it matters: Phase 3 evidence that a PSD-95 and α2-GABAA dual-target agent improves stroke outcomes, a field with few effective therapies.
Elevating the head to 30-40° for 72 hours after endovascular thrombectomy did not significantly improve 90 day functional outcomes versus a flat position of 0-10° in patients with anterior circulation large vessel occlusion stroke and successful reperfusion. Among 1368 adults at 67 Chinese stroke centres, the adjusted generalised odds ratio for lower disability on the modified Rankin scale was 1.12 (95% CI 0.97 to 1.29, P=0.14), and 90 day mortality was 18.3% versus 20.3% (P=0.18). Head position after thrombectomy appears to have little effect on recovery, though a modest clinically meaningful benefit remains possible.
Why it matters: It tests a common, zero-cost post-thrombectomy care decision in a large multicentre randomised trial.