Lancet neurology
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Randomized Controlled Trial
Safety and efficacy of continuous subcutaneous foslevodopa-foscarbidopa in patients with advanced Parkinson's disease: a randomised, double-blind, active-controlled, phase 3 trial.
Levodopa is the most effective symptomatic therapy for Parkinson's disease, but patients with advanced Parkinson's disease develop motor fluctuations with chronic oral levodopa therapy. Foslevodopa-foscarbidopa is a soluble formulation of levodopa and carbidopa prodrugs that is delivered as a 24-h/day continuous subcutaneous infusion, and we aimed to assess the safety and efficacy of this formulation in patients with advanced Parkinson's disease. ⋯ AbbVie.
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Randomized Controlled Trial Multicenter Study
Chronic remote ischaemic conditioning in patients with symptomatic intracranial atherosclerotic stenosis (the RICA trial): a multicentre, randomised, double-blind sham-controlled trial in China.
Intracranial atherosclerotic stenosis (ICAS) is one of the most common causes of stroke worldwide, and it is associated with a high risk of recurrent stroke with currently recommended treatments. We aimed to evaluate the effect of chronic remote ischaemic conditioning on prevention of ischaemic events in patients with symptomatic ICAS. ⋯ For the Chinese translation of the abstract see Supplementary Materials section.
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Randomized Controlled Trial Multicenter Study
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Risdiplam is an orally administered therapy that modifies pre-mRNA splicing of the survival of motor neuron 2 (SMN2) gene and is approved for the treatment of spinal muscular atrophy. The FIREFISH study is investigating the safety and efficacy of risdiplam in treated infants with type 1 spinal muscular atrophy versus historical controls. The primary endpoint of part 2 of the FIREFISH study showed that infants with type 1 spinal muscular atrophy attained the ability to sit without support for at least 5 s after 12 months of treatment. Here, we report on the safety and efficacy of risdiplam in FIREFISH part 2 over 24 months of treatment. ⋯ F Hoffmann-La Roche.
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Over the past decade, ocular imaging strategies have greatly advanced the diagnosis and follow-up of patients with optic neuropathies. Developments in optic nerve imaging have specifically improved the care of patients with papilloedema and idiopathic intracranial hypertension, inflammatory optic neuropathies, and compressive optic neuropathies. For example, optic nerve imaging with optical coherence tomography (OCT) is now widely used as an outcome measure in clinical trials of neurological disorders (eg, demyelinating diseases), and OCT findings could be informative of disease progression in patients with various neurodegenerative disorders (eg, Alzheimer's disease or Parkinson's disease). ⋯ Such multimodality imaging makes the diagnosis of optic neuropathies easier and provides objective information on optic nerve damage, which is useful for prognosis. Non-mydriatic ocular fundus cameras and OCT have become readily available in non-ophthalmic settings and could easily be implemented in neurological clinics and emergency departments, allowing for direct access to optic nerve imaging and enabling teleconsultations. In the future, these imaging studies could be used in association with artificial intelligence deep-learning systems, which are already transforming the field of ocular imaging.