Results 241 to 250 of about 2,331,898 (261)

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M. Tessier, Louis T. Dang   +7 more
wiley   +1 more source

Safety and Efficacy of GLP‐1 Receptor Agonists in Adults With Epilepsy, Obesity, and Type 2 Diabetes

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Managing obesity in patients with epilepsy is complicated by the weight‐gaining properties of essential antiseizure medications (ASMs) such as valproate and pregabalin. We evaluated the safety and efficacy of initiating glucagon‐like peptide‐1 receptor agonists (GLP‐1 RAs) in this population.
Hyoshin Son, Inyoung Hwang, Sang Won Lee, Yun Kim   +3 more
wiley   +1 more source

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Preventing Occupational Exposures to Antineoplastic Drugs in Health Care Settings

Ca-A Cancer Journal for Clinicians, 2006
M A McDiarmid, Thomas H Connor
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Outcome of Combining Epigenetic Drugs With Other Treatments in the Clinic

Medical Epigenetics, 2016
Paromita Raha
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Food and Drug Administration, Partner in Drug Development

Ca-A Cancer Journal for Clinicians, 2006
Jérôme W Yates, J W Yates
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Coexistence of Passive and Carrier-Mediated Processes in Drug Transport

Nature Reviews Drug Discovery, 2010
Kiyohiko Sugano, Li Di, Manfred Kansy
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Bio-Inspired, Bioengineered and Biomimetic Drug Delivery Carriers

Nature Reviews Drug Discovery, 2011
Jin-Wook Yoo, Dennis Discher, Samir Mitragotri   +2 more
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Mechanism of Action of Anticancer Drugs at a Cellular Level

Ca-A Cancer Journal for Clinicians, 1968
David A Karnofsky
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Palliation and Life Prolongation With Anticancer Drugs

Ca-A Cancer Journal for Clinicians, 1966
David A Karnofsky
exaly