Neurology, distilled.

To evaluate the safety, tolerability, and pharmacokinetics of vamorolone (2 or 6 mg/kg/d) in corticosteroid-naive boys with Duchenne muscular dystrophy aged 2-<4 years, with exploratory motor efficacy.

No deaths, no serious TEAEs, and no treatment discontinuations over 12 weeks; TEAEs more frequent with 6 mg/kg/d (90%) vs 2 mg/kg/d (70%), most commonly GI events and infections

To determine whether rituximab is noninferior to ocrelizumab for suppressing disease activity in adults with newly diagnosed relapsing multiple sclerosis.

Rituximab was noninferior to ocrelizumab for absence of new/enlarging T2 lesions from month 6-24 (92.2% vs 94.8%; risk difference -2.6 percentage points, 95% CI -9.4 to 4.3; P=0.03 for noninferiority)

To assess the safety and efficacy of oral levacetylleucine (N-acetyl-L-leucine) for the neurological manifestations of ataxia-telangiectasia in paediatric and adult patients.

First positive phase 3 RCT in ataxia-telangiectasia: levacetylleucine reduced SARA total score by −1·92 vs −0·14 with placebo (linear mixed model treatment effect −1·88, 95% CI −2·70 to −1·06; p<0·0001), exceeding the minimally important 1–1·5-point threshold.

To test whether DL-3-n-butylphthalide (NBP) improves cerebral blood flow in patients with chronic cerebral hypoperfusion from severe unilateral atherosclerotic stenosis of the internal carotid system.

NBP increased the proportion of patients achieving CBF amelioration at 12 weeks: 55.4% vs 43.9% (adjusted RR 1.32, 95% CI 1.08–1.61, p=0.006; unadjusted RR 1.26, 95% CI 1.04–1.54, p=0.020).

To compare short-term effects of levodopa-carbidopa-entacapone (LCE) versus levodopa-benserazide (LB) on plasma dopamine levels in early-stage unilateral Parkinson's disease.

No significant difference in plasma dopamine between LCE and LB at baseline (P=.744), 90 min (P=.704), or 180 min (P=.750)

To characterise plasma cytokine alterations during the ictal phase of CGRP-provoked idiopathic intracranial hypertension (IIH) headache attacks vs placebo, in a randomised, double-blind, two-way crossover provocation study.

IV CGRP (1.5 µg/min x 20 min) triggered a typical IIH headache attack in 7/16 women (44%); no participant developed headache after placebo

To assess the efficacy and safety of adjunctive tirofiban after successful endovascular reperfusion in patients with acute ischaemic stroke due to anterior-circulation large-vessel occlusion.

Functional independence (mRS 0-2) at 90 days: 49% (340/689) tirofiban vs 43% (299/691) placebo (absolute risk difference 6.1 percentage points, 95% CI 0.8-11.3, p=0.023; adjusted RR 1.15, 95% CI 1.03-1.27, p=0.0092)

Test whether adding biomarker-matched targeted therapy (erlotinib, everolimus or dasatinib) to standard radiotherapy improves overall survival in newly diagnosed, biopsy-proven diffuse intrinsic pontine glioma (DIPG).

Primary endpoint NOT met: median OS not improved over historical control in any arm (trial OS 11.1 mo vs historical 10.8 mo); trial stopped for futility.