Lancet neurology
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Comment Multicenter Study
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor neuron dysfunction. In this STR1VE-EU study, we aimed to evaluate the safety and efficacy of onasemnogene abeparvovec gene replacement therapy in infants with spinal muscular atrophy type 1, using broader eligibility criteria than those used in STR1VE-US. ⋯ Novartis Gene Therapies.
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The enormous societal and financial burden of Alzheimer's disease and related dementias requires the identification of risk factors and pathways to reduce dementia risk. Blood pressure (BP) management and control is one promising area, in which data have been inconclusive. ⋯ However, to date, the results do not concur on the optimal timing and target of BP lowering, and further study in diverse populations is needed. Given the long preclinical phase of dementia and data supporting the importance of BP control earlier in the lifecourse, long-term interventional and observational studies in ethnically and racially diverse populations, with novel imaging and blood-based biomarkers of neurodegeneration and vascular cognitive impairment to understand the pathophysiology, are needed to advance the field.
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There is an urgent unmet need for new therapies in amyotrophic lateral sclerosis. In a clinical study with healthy volunteers, levosimendan, a calcium sensitiser, was shown to improve neuromechanical efficiency and contractile function of the human diaphragm. We aimed to evaluate the safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis, with a focus on respiratory function. ⋯ Orion Corporation.