JAMA neurology
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Randomized Controlled Trial Multicenter Study
A randomized clinical trial of high-dosage coenzyme Q10 in early Parkinson disease: no evidence of benefit.
Coenzyme Q10 (CoQ10), an antioxidant that supports mitochondrial function, has been shown in preclinical Parkinson disease (PD) models to reduce the loss of dopamine neurons, and was safe and well tolerated in early-phase human studies. A previous phase II study suggested possible clinical benefit. ⋯ Coenzyme Q10 was safe and well tolerated in this population, but showed no evidence of clinical benefit.
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Multicenter Study
Predicting hematoma expansion after primary intracerebral hemorrhage.
Many clinical trials focus on restricting hematoma expansion following acute intracerebral hemorrhage (ICH), but selecting those patients at highest risk of hematoma expansion is challenging. ⋯ A 9-point prediction score for hematoma expansion was developed and independently validated. The results open a path for individualized treatment and trial design in ICH aimed at patients at highest risk of hematoma expansion with maximum potential for therapeutic benefit.
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Multicenter Study Observational Study
Frequency of hematoma expansion after spontaneous intracerebral hemorrhage in children.
Hematoma expansion is the only modifiable predictor of outcome in adult intracerebral hemorrhage; however, the frequency and clinical significance of hematoma expansion after childhood intracerebral hemorrhage are unknown. ⋯ Hematoma expansion occurs in children with intracerebral hemorrhage and may require urgent treatment. Repeat CT should be considered in children with either large hemorrhage or increased intracranial pressure.
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Multicenter Study Comparative Study
Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.
In Duchenne muscular dystrophy (DMD), the reading frame of an out-of-frame DMD deletion can be repaired by antisense oligonucleotide (AO)-mediated exon skipping. This creates a shorter dystrophin protein, similar to those expressed in the milder Becker muscular dystrophy (BMD). The skipping of some exons may be more efficacious than others. Patients with exon 44 or 45 skippable deletions (AOs in clinical development) have a less predictable phenotype than those skippable for exon 51, a group in advanced clinical trials. A way to predict the potential of AOs is the study of patients with BMD who have deletions that naturally mimic those that would be achieved by exon skipping. ⋯ Exon 44 or 45 skipping will likely yield lower levels of dystrophin than exon 51 skipping, although the resulting protein is functional enough to often maintain a mild BMD phenotype. Dystrophin transcript stability is an important indicator of dystrophin expression, and transcript instability in DMD compared with BMD should be explored as a potential biomarker of response to AOs. This study is beneficial for the planning, execution, and analysis of clinical trials for exon 44 and 45 skipping.
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Implementation of prehospital stroke triage is a public policy intervention that can have an immediate impact on acute stroke care in a region. OBJECTIVE To evaluate the impact that a citywide policy recommending prehospital triage of patients with suspected stroke to the nearest primary stroke center had on intravenous tissue plasminogen activator (tPA) use in Chicago, Illinois. ⋯ Implementation of a prehospital stroke triage policy in Chicago resulted in significant improvements in emergency medical services use and prenotification and more than doubled intravenous tPA use at primary stroke centers.