Lancet neurology
-
Randomized Controlled Trial
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We investigated the efficacy and safety of drisapersen, a 2'-O-methyl-phosphorothioate antisense oligonucleotide, given for 48 weeks. ⋯ GlaxoSmithKline, Prosensa Therapeutics BV (a subsidiary of Prosensa Holding NV).
-
Traumatic brain injury (TBI) is a major cause of morbidity and mortality. Concussion, a form of mild TBI, might be associated with long-term neurological symptoms. The effects of TBI and concussion are not restricted to cognition and balance. ⋯ Moderate and severe TBI might additionally lead to ocular motor palsies, optic neuropathies, and orbital pathologies. Vision-based testing is vital in the management of all forms of TBI and provides a sensitive approach for sideline or post-injury concussion screening. One sideline test, the King-Devick test, uses rapid number naming and has been tested in multiple athlete cohorts.
-
As preclinical Alzheimer's disease becomes a target for therapeutic intervention, the overlap between imaging abnormalities associated with typical ageing and those associated with Alzheimer's disease needs to be recognised. We aimed to characterise how typical ageing and preclinical Alzheimer's disease overlap in terms of β-amyloidosis and neurodegeneration. ⋯ US National Institute on Aging and Alexander Family Professorship of Alzheimer's Disease Research.
-
Biography Historical Article
Clifford Jack: biomarker curves and all that jazz.
-
Editorial Comment
Bringing forward the diagnosis of Alzheimer's disease.