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Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Neurology
January 2025
The Dubowitz Neuromuscular Centre, Developmental Neurosciences Department, University College London, Great Ormond Street Institute of Child Health, United Kingdom.
Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.
View Article and Find Full Text PDFTransient Hypoadrenocorticism in a 7-Week-Old Kitten.
J Am Anim Hosp Assoc
January 2025
From the Washington State University College of Veterinary Medicine, Pullman, Washington.
A 7 wk old female spayed domestic shorthair was evaluated for 3 days of lethargy and anorexia 1 wk after ovariohysterectomy. On presentation, she was weak, dehydrated, and hypotensive. Blood work showed azotemia, hyponatremia, hyperkalemia, hyperphosphatemia, and hyperproteinemia.
View Article and Find Full Text PDFThe brain's action-mode network.
Nat Rev Neurosci
January 2025
Mallinckrodt Institute of Radiology, Washington University School of Medicine, St. Louis, MO, USA.
The brain is always intrinsically active, using energy at high rates while cycling through global functional modes. Awake brain modes are tied to corresponding behavioural states. During goal-directed behaviour, the brain enters an action-mode of function.
View Article and Find Full Text PDFGuilu Erxian Jiao remodels dendritic spine morphology through activation of the hippocampal TRPC6-CaMKIV-CREB signaling pathway and suppresses fear memory generalization in rats with post-traumatic stress disorder.
J Ethnopharmacol
December 2024
Integrative Medicine Research Center, School of Basic Medical Sciences, Guangzhou University of Chinese Medicine, Outer Ring East Road No. 232, Higher Education Mega Center, Guangzhou, 510006, China. Electronic address:
Ethnopharmacological Relevance: Guilu Erxian Jiao (GLEXJ) is a renowned traditional Chinese herbal formula used to tonify the kidney. It is employed to treat psychiatric disorders, and alleviate memory impairment, cognitive dysfunction, and behavioral disorders. Modern pharmacological studies have demonstrated GLEXJ's ability to significantly inhibit the fear response in post-traumatic stress disorder (PTSD) and facilitate the extinction of fear memory.
View Article and Find Full Text PDFComparing the efficacy of oral apremilast, intralesional corticosteroids, and their combination in patients with patchy alopecia areata: a randomized clinical controlled trial.
Arch Dermatol Res
December 2024
Department of Dermatology, Venereology and Leprology, Mahatma Gandhi Medical College and Hospital, Jaipur, Rajasthan, India.
Introduction: Alopecia areata (AA) is a chronic, immune-mediated inflammatory disorder characterized by nonscarring hair loss. The management of AA poses challenges due to its unpredictable course and variable response to treatment. In this comparative study, we evaluated the efficacy and safety of oral apremilast, intralesional corticosteroids (ILC) and a combination of both in patients with patchy AA.
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