Results 1 to 10 of about 56,380 (164)

Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia

Frontiers in Oncology, 2022
Blocking the pyrimidine nucleotide de novo synthesis pathway by inhibiting dihydroorotate dehydrogenase (DHODH) results in the cell cycle arrest and/or differentiation of rapidly proliferating cells including activated lymphocytes, cancer cells, or ...
Arthur Branstrom, Liangxian Cao, Bansri Furia, Christopher Trotta, Marianne Santaguida, Jason D. Graci, Joseph M. Colacino, Balmiki Ray, Wencheng Li, Josephine Sheedy, Anna Mollin, Shirley Yeh, Ronald Kong, Richard Sheridan, John D. Baird, Kylie O’Keefe, Robert Spiegel, Elizabeth Goodwin, Suzanne Keating, Marla Weetall   +19 more
doaj   +1 more source

A pharmacokinetic drug–drug interaction study between rosuvastatin and emvododstat, a potent anti‐SARS‐CoV‐2 (COVID‐19) DHODH (dihydroorotate dehydrogenase) inhibitor

Pharmacology Research & Perspectives, 2023
A therapeutic agent that targets both viral replication and the hyper‐reactive immune response would offer a highly desirable treatment for severe acute respiratory syndrome coronavirus 2 (SARS‐CoV‐2; COVID‐19) management.
Terri L. Morton, Oscar L. Laskin, Diksha Kaushik, Lucy Lee, Jiyuan Ma, Allan Kristensen, Kylie O'Keefe, Lee Golden, Matthew Klein, Ronald Kong   +9 more
doaj   +1 more source

Small molecule splicing modifiers with systemic HTT-lowering activity

Nature Communications, 2021
Here the authors describe the discovery of a class of small molecule splicing modifiers which are orally bioavailable, cross the blood-brain barrier, and lower levels of huntingtin in a mouse model of Huntington’s disease (HD).
Anuradha Bhattacharyya, Christopher R. Trotta, Jana Narasimhan, Kari J. Wiedinger, Wencheng Li, Kerstin A. Effenberger, Matthew G. Woll, Minakshi B. Jani, Nicole Risher, Shirley Yeh, Yaofeng Cheng, Nadiya Sydorenko, Young-Choon Moon, Gary M. Karp, Marla Weetall, Amal Dakka, Vijayalakshmi Gabbeta, Nikolai A. Naryshkin, Jason D. Graci, Thomas Tripodi, Amber Southwell, Michael Hayden, Joseph M. Colacino, Stuart W. Peltz   +23 more
doaj   +1 more source

Experiencias Pedagógicas entre Discapacidad, Infancia y Matemáticas

Educación y Ciencia, 2023
El presente artículo pretende identificar cuáles son las condiciones que fortalecen el reconocimiento numérico de una sola cifra, base del pensamiento matemático en una estudiante de nueve años que presenta Discapacidad Intelectual (DI) y cursa tercero ...
Marvin Amaya-Gil, Angie Patiño-Benavides, Julieth Marín-Pineda   +2 more
doaj   +1 more source

A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy

Journal of Patient-Reported Outcomes, 2021
Background Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory ...
Kate Williams, Ian Davidson, Mark Rance, Katharina Buesch, Sarah Acaster   +4 more
doaj   +1 more source

Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model

Journal of Patient-Reported Outcomes, 2021
Background Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory ...
Kate Williams, Ian Davidson, Mark Rance, Axel Boehnke, Katharina Buesch, Sarah Acaster   +5 more
doaj   +1 more source

In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren

Pharmacology Research & Perspectives, 2020
Ataluren promotes ribosomal readthrough of premature termination codons in mRNA which result from nonsense mutations. In vitro studies were performed to characterize the metabolism and enzyme kinetics of ataluren and its interaction potential with CYP ...
Ronald Kong, Jiyuan Ma, Seongwoo Hwang, Young‐Choon Moon, Ellen M. Welch, Marla Weetall, Joseph M. Colacino, Neil Almstead, John Babiak, Elizabeth Goodwin   +9 more
doaj   +1 more source

In vitro cytochrome P450‐ and transporter‐mediated drug interaction potential of 6β‐hydroxy‐21‐desacetyl deflazacort—A major human metabolite of deflazacort

Pharmacology Research & Perspectives, 2021
6β‐Hydroxy‐21‐desacetyl deflazacort (6β‐OH‐21‐desDFZ) is a major circulating but not biologically active metabolite of deflazacort (DFZ). In vitro studies were performed to evaluate cytochrome P450 (CYP)‐ and transporter‐mediated drug interaction ...
Jiyuan Ma, Brian Beers, Ravi Manohar, Stephen Roe, Joseph M. Colacino, Ronald Kong   +5 more
doaj   +1 more source

Ribonucleotide reductase, a novel drug target for gonorrhea

eLife, 2022
Antibiotic-resistant Neisseria gonorrhoeae (Ng) are an emerging public health threat due to increasing numbers of multidrug resistant (MDR) organisms. We identified two novel orally active inhibitors, PTC-847 and PTC-672, that exhibit a narrow spectrum ...
Jana Narasimhan, Suzanne Letinski, Stephen P Jung, Aleksey Gerasyuto, Jiashi Wang, Michael Arnold, Guangming Chen, Jean Hedrick, Melissa Dumble, Kanchana Ravichandran, Talya Levitz, Chang Cui, Catherine L Drennan, JoAnne Stubbe, Gary Karp, Arthur Branstrom   +15 more
doaj   +1 more source

Metabolite V, an epoxide species is a minor circulating metabolite in humans following a single oral dose of deflazacort

Pharmacology Research & Perspectives, 2020
Deflazacort (Emflaza) was approved in the United States in 2017 for the treatment of the Duchenne muscular dystrophy in patients aged 2 years and older.
Ronald Kong, Jiyuan Ma, Brian Beers, Diksha Kaushik, E Lin, Elizabeth Goodwin, Joseph Colacino, Francesco Bibbiani   +7 more
doaj   +1 more source