Results 51 to 60 of about 485,740 (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

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

Original drugs approved by the Food and Drug Administration (Center for Drug Evaluation and Research) in 2024

Фармация и фармакология (Пятигорск)
The U.S. Food and Drug Administration (FDA), in particular the Center for Drug Evaluation and Research (CDER), plays a key role in ensuring the safety, efficacy, and innovation of medicines entering the U.S. market, and then the world.
D. V. Kurkin, N. A. Osadchenko, A. R. Makarova, D. A. Galkina, D. A. Bakulin, O. V. Shatalova, A. V. Strygin, V. I. Petrov, O. V. Marincheva, Yu. V. Gorbunova, Yu. A. Kolosov, A. V. Zaborovsky, D. V. Yunina, K. N. Koryanova, E. I. Morkovin, M. A. Dzhavakhyan, V. I. Zvereva, R. V. Drai, I. E. Makarenko, A. S. Shuvaeva   +19 more
doaj   +1 more source

Доказана генетическая предрасположенность к хронической боли

Медицинский совет, 2014
Обнаружены гены, определяющие чувствительность человека к хронической боли, сообщает Medical News Today по материалам ежегодной конференции Американской академии неврологии в Филадельфии.
Editorial Article
doaj   +1 more source

Building a Framework for Sexual and Reproductive Health Care in the Rheumatology Context: Content and Approaches

Arthritis Care &Research, EarlyView.
People with systemic autoimmune and rheumatic diseases (SARDs) are at higher risk than the general population of experiencing adverse pregnancy and perinatal outcomes such as preeclampsia, intrauterine growth restriction, and maternal and/or fetal death.
Mehret Birru Talabi, Sonya Borrero
wiley   +1 more source

A whole-cell pertussis vaccine engineered to elicit reduced reactogenicity protects baboons against pertussis challenge

mSphere
Whole-cell pertussis (wP) vaccines introduced in the 1940s led to a dramatic reduction of pertussis incidence and are still widely used in low- and middle-income countries (LMICs) worldwide.
Parul Kapil, Yihui Wang, Kelsey Gregg, Lindsey Zimmerman, Damaris Molano, Jonatan Maldonado Villeda, Peter Sebo, Tod J. Merkel   +7 more
doaj   +1 more source

В целом пациенты удовлетворены лечением послеоперационных болей

Медицинский совет, 2014
Пациенты, принимающие непосредственное участие в принятии решений, связанных с лечением послеоперационных болей, в большей степени удовлетворены проводимой терапией.
Editorial Article
doaj   +1 more source

Orphanage at the FDA [PDF]

Journal of Cardiovascular Translational Research, 2010
In the late 1970s and early 1980s, social and political forces gave birth to a new movement to serve the needs of people with rare diseases, a movement that has only accelerated with the penetrance of internet connectivity. The FDA's Office of Orphan Products Development (OOPD) was established in 1982 to promote the development of products that ...
openaire   +2 more sources

From Adult to Adolescent: Alignment in Clinical Trials and Outcomes in Axial Spondyloarthritis

Arthritis Care &Research, EarlyView.
Spondyloarthritis (SpA) is a group of chronic inflammatory diseases encompassing axial and peripheral forms, with up to 20% of patients developing symptoms before age 16. Despite this substantial pediatric burden, treatment options for juvenile‐onset SpA (JSpA), particularly those with axial disease (axJSpA), remain limited.
Pamela F. Weiss, Judith A. Smith, Robert A. Colbert, Erin Treemarcki, Michael H. Weisman, Walter P. Maksymowych, Hermine I. Brunner, for the Childhood Arthritis and Rheumatology Research Alliance, Spondyloarthritis Research and Treatment Network, and Pediatric Rheumatology Collaborative Study Group Advisory Council   +9 more
wiley   +1 more source

Clinical Validation of Artificial Intelligence (AI)‐based Cartilage Segmentation Predicting Knee Replacement

Arthritis Care &Research, Accepted Article.
Objective For cartilage morphology to serve as a scalable endpoint in clinical trials, analyses should be performed automatically without human interaction. To clinically validate artificial intelligence (AI)‐based analysis, we studied cartilage loss from MRI prior to knee replacement.
Felix Eckstein, David J. Hunter, C. Kent Kwoh, Wolfgang Wirth   +3 more
wiley   +1 more source