Results 51 to 60 of about 160,551 (221)

The immunophilin protein FKBPL and its peptide derivatives are novel regulators of vascular integrity and inflammation via NF-κB signaling

bioRxiv, 2021
A breakdown in vascular integrity and excessive inflammation are hallmarks of serious pathological conditions including sepsis, acute respiratory distress syndrome (ARDs) and most recently, severe COVID-19.
Stephanie Annett, S. Spence, Carolina D. Garciarena, C. Campbell, M. Dennehy, Clive Drakeford, Jacqueline C Y Lai, J. Dowling, G. Moore, A. Yakkundi, Amy Short, D. Sharpe, F. Furlong, J. O’Donnell, G. Cavalleri, S. Kerrigan, I. Tikhonova, Pauline Johnson, A. Kissenpfennig, T. Robson   +19 more
semanticscholar   +1 more source

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

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

Revolutionizing Drug Delivery: The Impact of Advanced Materials Science and Technology on Precision Medicine

Pharmaceutics
Recent progress in material science has led to the development of new drug delivery systems that go beyond the conventional approaches and offer greater accuracy and convenience in the application of therapeutic agents.
Mohamed El-Tanani, Shakta Mani Satyam, Syed Arman Rabbani, Yahia El-Tanani, Alaa A. A. Aljabali, Ibrahim Al Faouri, Abdul Rehman   +6 more
doaj   +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

OR02-2 CX-4945 as a Potential Drug for Adrenocortical Carcinoma That Induces Multiple Exon-Skipping and Circular RNA of NR5A1

Journal of the Endocrine Society, 2019
Orphan nuclear receptor NR5A1 (also called adrenal 4-binding protein/steroidogenic factor-1) is the major regulator of differentiation and function of the adrenal cortex.
Tomoko Tanaka, K. Ohe, T. Nomiyama, T. Yanase   +3 more
semanticscholar   +1 more source

Effects of Secukinumab on Enthesiophyte and Erosion Progression in Psoriatic Arthritis: A One‐Year Double‐Blind, Randomized, Placebo‐Controlled Trial Using High‐Resolution Peripheral Quantitative Computed Tomography

Arthritis &Rheumatology, EarlyView.
Objective This study aimed to ascertain the effect of secukinumab on erosion and enthesiophyte progression in psoriatic arthritis (PsA) by using high‐resolution peripheral quantitative computed tomography (HR‐pQCT). Methods This was a phase 4, double‐blind, randomized, placebo‐controlled trial.
Yingzhao Jin, Isaac T. Cheng, Ho So, Billy T. Lai, Shirley K. Ying, Kitty Y. Kwok, James Griffith, Vivian Hung, Cheuk‐Chun Szeto, Jack J. Lee, Ling Qin, Lai‐Shan Tam   +11 more
wiley   +1 more source

New Insights in Cushing Disease Treatment With Focus on a Derivative of Vitamin A

Frontiers in Endocrinology, 2018
Cushing’s disease (CD) is an endocrine disorder originated by a corticotroph tumor. It is linked with high mortality and morbidity due to chronic hypercortisolism. Treatment goals are to control cortisol excess and achieve long-term remission, therefore,
Mariana Fuertes, Julieta Tkatch, Josefina Rosmino, Leandro Nieto, Mirtha Adriana Guitelman, Eduardo Arzt, Eduardo Arzt   +6 more
doaj   +1 more source

The outcome of more than 3 decades of orphan drug development in alpha-1-antitypsin deficiency – a cross sectional analysis of the FDA Orphan Drug Product designation database

, 2022
Abstract Introduction Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into the status of FDA orphan drug approvals and designations for compounds ...
Franziska C Trudzinski, Maria Ada Presotto, Emanuel Buck, Felix J. Herth, Markus Ries   +4 more
openaire   +1 more source

Activin A–Activated ALK4 Induces Pathogenic Th17‐Involved Endothelial–Mesenchymal Transition in Systemic Lupus Erythematosus–Associated Pulmonary Arterial Hypertension

Arthritis &Rheumatology, EarlyView.
Objective Autoimmune diseases, such as systemic lupus erythematosus (SLE), are associated with pulmonary arterial hypertension (PAH), a condition that can lead to heart failure. However, whether T cells also contribute to the occurrence of PAH in SLE has not been clarified.
Shuliang Jing, Junyan Qian, Hongjie Ying, Pei Mao, Mingxin Yao, Zhihong Wu, Harm J. Bogaard, Lie Wang, Mengtao Li, Jun Yang   +9 more
wiley   +1 more source