Results 21 to 30 of about 43,399 (123)

Repurposing With Purpose: Treatment of Bachmann–Bupp Syndrome With Eflornithine and Implications for Other Polyaminopathies

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Rare diseases impact approximately 1 in 10 people worldwide, and yet, less than 5% of all rare diseases currently have an approved treatment option available. This is due to many challenges unique to rare diseases, including small, diverse patient populations, the cost of drug development that is not proportionate to the number of patients who
Caleb P. Bupp, Elizabeth A. VanSickle, Julianne Michael, Chad R. Schultz, Kelly Nguyen, Melissa Hoefer, Surender Rajasekaran, André S. Bachmann   +7 more
wiley   +1 more source

Endocannabinoid system and anticancer properties of cannabinoids [PDF]

, 2016
Cannabinoids impact human body by binding to cannabinoids receptors (CB1 and CB2). The two main phytocannabinoids are Δ9-tetrahydrocannabinol (THC) and cannabidiol (CBD).
Nowak, Agnieszka, Słomski, Ryszard, Zeyland, Joanna, Śledziński, Paweł   +3 more
core   +1 more source

Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies

Annals of Neurology, EarlyView.
Autosomal recessive cerebellar ataxias are disabling neurodegenerative genetic conditions affecting balance and coordination. Advancements in genomic testing have improved diagnosis, leading to a new focus on the development of targeted precision therapeutics addressing cellular, biochemical, and genetic disease mechanisms with a resulting emphasis on ...
Brent L. Fogel, Thomas Klopstock, David R. Lynch, Francesca Maltecca, Mayank Verma, Berge A. Minassian, Frances M. Platt, Débora Farina Gonçalves, Hélène Puccio, Andreas Roos, Matthis Synofzik   +10 more
wiley   +1 more source

An expanding range of targets for kynurenine metabolites of tryptophan [PDF]

, 2013
The kynurenine pathway of tryptophan metabolism accounts for most of the tryptophan that is not committed to protein synthesis and includes compounds active in the nervous and immune systems.
Darlington, L. Gail, Stone, Trevor W., Stoy, Nicholas   +2 more
core   +1 more source

Roadmap to Nanomedical Applications: Nanotoxicology and In Vitro Guidelines for Lanthanide‐Doped Luminescence Nanothermometers

Advanced NanoBiomed Research, EarlyView.
Lanthanide‐based luminescence nanothermometry holds multifaceted potential for nanomedical applications, yet despite significant research advances, clinical translation of the engineered nanomaterials is still controversial and widely disputed. This is related to crucial nanotoxicological aspects that must be addressed at the preclinical level ...
Simona Premcheska, Andre G. Skirtach, Anna M. Kaczmarek   +2 more
wiley   +1 more source

T Helper Cells: The Modulators of Inflammation in Multiple Sclerosis [PDF]

, 2020
Multiple sclerosis (MS) is a chronic neurodegenerative disease characterized by the progressive loss of axonal myelin in several areas of the central nervous system (CNS) that is responsible for clinical symptoms such as muscle spasms, optic neuritis ...
Amormino, Carola, Frascolla, Simone, Kunkl, Martina, Tuosto, Loretta, Volpe, Elisabetta   +4 more
core   +1 more source

Mechanochemical Strategies Applied to the Late‐Stage Modifications of Pharmaceutically Active Compounds

Angewandte Chemie International Edition, Accepted Article.
This review explores the potential of mechanochemistry in the late‐stage modification of active pharmaceutical ingredients (APIs), offering a comprehensive analysis of methods designed to transform structurally complex molecular scaffolds. By examining the scope, efficiency, and mechanistic aspects of these approaches, the review highlights protocols ...
Johanna Templ, Lars Borchardt
wiley   +1 more source

Access to Life-Saving Medicines and Intellectual Property Rights: An Ethical Assessment [PDF]

, 2011
© 2011 Cambridge University Press. Online edition of the journal is available at http://journals.cambridge.org/action/displayJournal?jid=CQHDying before one’s time has been a prominent theme in classic literature and poetry.
Aquinas, Aquinas, Aquinas, Brown, DORIS SCHROEDER, Finnis, Jentzsch, Leisinger, Locke, Mamunes, May, Nozick, Pang, PETER SINGER, Pogge, Pogge, Rawls, Rifkin, Schroeder, Selgelid, Shelley   +20 more
core   +2 more sources

Metabolic Stress Expands Polyfunctional, Proinflammatory Th17 Cells in Patients With Psoriatic Arthritis for Whom There is Interleukin‐23–Independent Interleukin‐17 Production

Arthritis &Rheumatology, EarlyView.
Objective Genetic associations and blockade of the interleukin (IL)‐23/IL‐17 axis with monoclonal antibodies support a role for this pathway in patients with psoriatic arthritis (PsA). This study examines the requirement of IL‐23 for IL‐17 production and the role of the metabolic microenvironment in the expansion of Th17‐derived cells in patients with ...
Carmel B. Stober, Louise Ellis, Jane C. Goodall, Marc Veldhoen, J. S. Hill Gaston   +4 more
wiley   +1 more source

Three classes of ligands each bind to distinct sites on the orphan G protein-coupled receptor GPR84 [PDF]

, 2017
Medium chain fatty acids can activate the pro-inflammatory receptor GPR84 but so also can molecules related to 3,3′-diindolylmethane. 3,3′-Diindolylmethane and decanoic acid acted as strong positive allosteric modulators of the function of each other and
De Vos, Steve, Gosmini, Romain, Hudson, Brian D., Jenkins, Laura, Labéguère, Frédéric, Mahmud, Zobaer Al, Milligan, Graeme, Tikhonova, Irina G., Ulven, Trond   +8 more
core   +2 more sources