Lancet neurology
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Randomized Controlled Trial Multicenter Study
Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial.
Spinal and bulbar muscular atrophy is an X-linked neuromuscular disease caused by CAG repeat expansion in the androgen receptor gene. Patients with this disease have low concentrations of insulin-like growth factor-1 (IGF-1), and studies of overexpression and administration of IGF-1 showed benefit in a transgenic model; thus the IGF-1 pathway presents as a potential treatment target. We assessed safety, tolerability, and preliminary efficacy of BVS857, an IGF-1 mimetic, in patients with spinal and bulbar muscular atrophy. ⋯ Novartis Pharmaceuticals and the US National Institutes of Health.
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Cardiac complications are a frequent medical problem during the first few days after an ischaemic stroke, and patients present with a broad range of symptoms including myocardial injury, cardiac dysfunction, and arrhythmia, with varying overlap between these three conditions. Evidence from clinical and neuroimaging studies and animal research suggests that these cardiac disturbances share the same underlying mechanisms. Although the exact cascade of events has yet to be elucidated, stroke-induced functional and structural alterations in the central autonomic network, with subsequent dysregulation of normal neural cardiac control, are the assumed pathophysiology. ⋯ These stroke-associated cardiac alterations can be summarised as a distinct so-called stroke-heart syndrome. Independent cohort studies have shown a strong association between this syndrome and unfavourable short-term prognosis; however, long-term consequences, including secondary cardiac events and death, are less well described and specific therapeutic targets are scarce. An integrated view of stroke-heart syndrome will offer opportunities to expedite research and inform clinical decision making.