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Department of Radiology and Medical Imaging, King Saud University Medical City, King Saud University, Riyadh, KSA.
Background: Multiple sclerosis (MS) is one of the most common disabling central nervous system diseases affecting young adults. Magnetic resonance imaging (MRI) is an essential tool for diagnosing and following up multiple sclerosis. Over the years, many MRI techniques have been developed to improve the sensitivity of MS disease detection.
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Clin Transl Med
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Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Background: Fabry disease is an X-linked lysosomal storage disorder due to a deficiency of α-galactosidase A (α-gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α-gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells.
View Article and Find Full Text PDFSystematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients with X-Linked Hypophosphatemia.
J Clin Endocrinol Metab
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Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada.
Objective: To examine the evidence addressing the management of X-linked hypophosphatemia (XLH) in children to inform treatment recommendations.
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Harnessing the Sulfur-for-Oxygen Shift: A Magic Bullet for Dynamic Photophysical and Anticancer Activities of Indole-Barbituric Acid Construct.
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IIT Roorkee: Indian Institute of Technology Roorkee, Chemistry, Department of Chemistry, 247667, Roorkee, INDIA.
The development of small molecule-based drugs emerged as a cornerstone of modern drug discovery. Structural activity relationship (SAR) studies in medicinal chemistry are crucial for lead optimization, where a subtle change in the substituent can significantly alter its binding affinity with the biological target. Herein, a highly efficient single-atom substitution (SAS) approach has been developed, where sulfur for oxygen strategy is utilized as a powerful molecular editing technique to identify N-vinyl Indole-thiobarbituric acid (6a) as a novel small molecule-based scaffold with tunable photophysical and antiproliferative activities.
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Pediatr Blood Cancer
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Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
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