Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.

Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a shortened dystrophin retaining key functional domains of the wild-type protein. This Phase 2, double-blind, two-part (48 weeks per part) crossover study (SRP-9001-102 [Study 102]; NCT03769116) evaluated delandistrogene moxeparvovec in patients, aged ≥4 to <8 years with Duchenne muscular dystrophy. Primary endpoints (Part 1) were change from baseline (CFBL) in SRP-9001 dystrophin expression (Week 12), by Western blot, and in North Star Ambulatory Assessment (NSAA) score (Week 48).

Interfragmentary strain measurement post-fixation to guide intraoperative decision making: a narrative review.

Purpose: Interfragmentary strain influences whether a fracture will undergo direct and indirect fracture healing. Orthopedic trauma surgeons modulate strain and create optimal biomechanical environments for specific fracture patterns using fixation constructs. However, objective intraoperative interfragmentary strain measurement does not currently inform fixation strategy in common practice.

Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy.

Deflazacort and prednisone/prednisolone are the current standard of care for patients with Duchenne muscular dystrophy (DMD) based on evidence that they improve muscle strength, improve timed motor function, delay loss of ambulation, improve pulmonary function, reduce the need for scoliosis surgery, delay onset of cardiomyopathy, and increase survival. Both have been used off-label for many years (choice dependent on patient preference, cost, and geographic location) before FDA approval of deflazacort for DMD in 2017. In this review, we compare deflazacort and prednisone/prednisolone in terms of their key pharmacological features, relative efficacy, and safety profiles in patients with DMD.

3D/4D Reconstruction and Quantitative Total Body Imaging.

Total-body PET image reconstruction follows a similar procedure to the image reconstruction process for standard whole-body PET scanners. One unique aspect of total-body imaging is simultaneous coverage of the entire human body, which makes it convenient to perform total-body dynamic PET scans. Therefore, four-dimensional dynamic PET reconstruction and parametric imaging are of great interest in total-body imaging.

Cardioembolic Ischemic Stroke Gene Expression Fingerprint in Blood: a Systematic Review and Verification Analysis.

An accurate etiological classification is key to optimize secondary prevention after ischemic stroke, but the cause remains undetermined in one third of patients. Several studies pointed out the usefulness of circulating gene expression markers to discriminate cardioembolic (CE) strokes, mainly due to atrial fibrillation (AF), while only exploring them in small cohorts. A systematic review of studies analyzing high-throughput gene expression in blood samples to discriminate CE strokes was performed.

Gating by inhibition during top-down control of willed attention.

In natural settings, the control of attention may be influenced both by external information as well as internal decision-making processes driven by intent (e.g. free will).

Scaling of the surface vasculature on the human placenta.

The networks of veins and arteries on the chorionic plate of the human placenta are analyzed in terms of Voronoi cells derived from these networks. Two groups of placentas from the United States are studied: a population cohort with no prescreening, and a cohort from newborns with an elevated risk of developing autistic spectrum disorder. Scaled distributions of the Voronoi cell areas in the two cohorts collapse onto a single distribution, indicating common mechanisms for the formation of the complete vasculatures, but which have different levels of activity in the two cohorts.

An Exploratory Human Laboratory Experiment Evaluating Vaporized Cannabis in the Treatment of Neuropathic Pain From Spinal Cord Injury and Disease.

Unlabelled: Using 8-hour human laboratory experiments, we evaluated the analgesic efficacy of vaporized cannabis in patients with neuropathic pain related to injury or disease of the spinal cord, most of whom were experiencing pain despite traditional treatment. After obtaining baseline data, 42 participants underwent a standardized procedure for inhaling 4 puffs of vaporized cannabis containing either placebo, 2.9%, or 6.

Current and future alternatives to warfarin for the prevention of stroke in atrial fibrillation.

Atrial fibrillation (AF) is the most common sustained arrhythmia in clinical practice and is associated with a nearly 5-fold increase in the risk of stroke. Warfarin has been the cornerstone of treatment to reduce stroke risk in AF patients for decades. Although effective in preventing thrombosis, warfarin is difficult to manage and is associated with a 1% to 7% yearly risk of major hemorrhage.

Optimal treatment of femoral neck fractures according to patient's physiologic age: an evidence-based review.

For decades, the basic tenets of managing displaced femoral neck fractures have not changed, but the optimal treatment choice continues to be highly debated. The contemporary controversies associated with the treatment principles of displaced femoral neck fractures are distinct between young and old patients and are considered individually in this article about the current evidence. Although fixation constructs all seem to have similar complication rates, there is increasing evidence suggesting that total hip replacement improves patient functional outcomes for healthy, independent, elderly patients compared with hemiarthroplasty and should be considered as the treatment of choice for these patients.

Safety and efficacy of renal artery stenting following suboptimal renal angioplasty for de novo and restenotic ostial lesions: results from a nonrandomized, prospective multicenter registry.

Purpose: This registry was designed to evaluate the Bridge Extra Support renal stent system (Medtronic, Santa Rosa, California) in the treatment of renal artery ostial lesions following suboptimal percutaneous transluminal renal angioplasty (PTRA).

Materials And Methods: This consecutive, nonrandomized, prospective, multicenter registry enrolled 188 patients between April 1999 and May 2002 with single de novo or restenotic renal artery ostial lesions (>or=70% stenosis) who underwent implantation of a balloon-expandable stent immediately following unsuccessful PTRA. Primary safety endpoints were major adverse clinical events (MACE) at 30 days and 9-12 months.

Colonoscopy findings in end-stage liver disease patients with incidental CT colonic wall thickening.

Objective: Colonic wall thickening may occur in patients with end-stage liver disease. This study was conducted to correlate colonoscopy with CT-detected colonic wall thickening in this group of patients to assess their radiologic and clinical relevance.

Conclusion: Our results suggest that CT findings of colonic wall thickening in end-stage liver disease should be considered benign, and colonoscopy is unnecessary for the evaluation of malignancy or colitis unless it is clinically indicated.