Neurology
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Randomized Controlled Trial Multicenter Study
Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS.
To examine the safety and tolerability as well as the preliminary efficacy of arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS). ⋯ This study provides Class II evidence that arimoclomol is safe and well-tolerated at a dosage of 200 mg tid for up to 12 months. The study lacked the precision to conclude, or to exclude, an important therapeutic benefit of arimoclomol.
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To investigate whether the clinical characteristics of patients with hemiplegic migraine with and without autosomal dominant mutations in CACNA1A, ATP1A2, or SCN1A differ, and whether the disease may be caused by mutations in other genes. ⋯ Most patients with hemiplegic migraine without a mutation in CACNA1A, ATP1A2, or SCN1A display a mild phenotype that is more akin to that of common (nonhemiplegic) migraine. A major fourth autosomal dominant gene for hemiplegic migraine remains to be identified. Our observations might guide physicians in selecting patients for mutation screening and in providing adequate genetic counseling.
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Multicenter Study
Racial disparities in refusal of stroke thrombolysis in Chicago.
To evaluate race differences in tissue plasminogen activator (tPA) refusal among eligible patients with acute ischemic stroke (AIS) in Chicago. ⋯ Among tPA-eligible patients with AIS in Chicago, over 7% refused tPA. Refusal was more common in black patients and accounted for the apparent lower rates of tPA use in black vs nonblack patients. Further research is needed to understand barriers to consent and overcome race-ethnic disparities in tPA treatment for AIS.
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Meta Analysis
Circulating insulin-like growth factors and Alzheimer disease: A mendelian randomization study.
To examine whether genetically predicted variation in circulating insulin-like growth factor 1 (IGF1) or its binding protein, IGFBP3, are associated with risk of Alzheimer disease (AD), using a mendelian randomization study design. ⋯ These findings suggest that circulating IGF1 and IGFBP3 are not important determinants of AD risk. FOXO3 function may influence AD development via pathways that are independent of IGF signaling (i.e., pleiotropic actions).
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To assess the relative risk of oral clefts associated with maternal use of high and low doses of topiramate during the first trimester for epilepsy and nonepilepsy indications. ⋯ The increased risk of oral clefts associated with use of topiramate early in pregnancy was more pronounced in women with epilepsy, who used higher doses.