OUTCOME OF FOUR PATIENTS WITH OSTEONECROSIS AFTER ONE-YEAR PAMIDRONATE TREATMENT.

Context: Osteonecrosis (ON) is bone death caused by inadequate blood supply and its optimal management remains uncertain.

Objective: We describe the outcomes of BP (pamidronate) treatment in our patients.

Design: Data regarding clinical, laboratory, magnetic resonance imaging (MRI) studies, and bone mineral density measurements (BMD) were recorded before and one year after treatment (reevaluation).

Prediction model for severe vesicoureteral reflux in children with urinary tract infection and/or hydronephrosis.

Background: As voiding cystourethrography is invasive and exposes to radiation and urinary tract infection (UTI), identifying only high-grade reflux is important. We aimed to identify clinical, laboratory and imaging variables associated with high-grade primary reflux in children presenting with UTIs and/or urinary tract dilatation and develop a prediction model for severe reflux.

Methods: Data of children who underwent voiding cystourethrography due to UTI and/or urinary tract dilatation were retrospectively analyzed for demographic, clinical and imaging findings.

Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy.

Background: Spinal muscular atrophy (SMA) is a motor neuron disease caused by mutations in the SMN1 gene. Nusinersen, an antisense oligonucleotide, has been shown to improve motor function in SMA patients. However, concerns regarding its renal safety remain as previous studies have linked similar treatments to renal toxicity.

Reduced PI3K(p110α) induces atrial myopathy, and PI3K-related lipids are dysregulated in athletes with atrial fibrillation.

Background: Elucidating mechanisms underlying atrial myopathy, which predisposes individuals to atrial fibrillation (AF), will be critical for preventing/treating AF. In a serendipitous discovery, we identified atrial enlargement, fibrosis, and thrombi in mice with reduced phosphoinositide 3-kinase (PI3K) in cardiomyocytes. PI3K(p110α) is elevated in the heart with exercise and is critical for exercise-induced ventricular enlargement and protection, but the role in the atria was unknown.

An optimized plasmalogen modulating dietary supplement provides greater protection in a male than female mouse model of dilated cardiomyopathy.

We previously reported that plasmalogens, a class of phospholipids, were decreased in a setting of dilated cardiomyopathy (DCM). Plasmalogen levels can be modulated via a dietary supplement called alkylglycerols (AG) which has demonstrated benefits in some disease settings. However, its therapeutic potential in DCM remained unknown.