Results 41 to 50 of about 88,754 (289)

First insights into the phylogenetic diversity of Mycobacterium tuberculosis in Nepal [PDF]

, 2012
BACKGROUND: Tuberculosis (TB) is a major public health problem in Nepal. Strain variation in Mycobacterium tuberculosis may influence the outcome of TB infection and disease. To date, the phylogenetic diversity of M.
A Sandgren, AE Hirsh, AN Malik, BC de Jong, Bhagwan Maharjan, Bhawana Shrestha, Bijaya Malla, C Demay, CB Holmes, CB Uwizeye, Christophe Sola, D Stucki, David Stucki, E Lasunskaia, E Svensson, ES Click, F Brossier, FA Drobniewski, G Thwaites, H Dong, H Getahun, H Han, HY Dou, I Comas, I Parwati, J Kamerbeek, JK Bonfield, JR Sun, Julia Feldmann, K Brudey, K Brudey, L Baker, L Fenner, Lukas Fenner, M Caws, M Connolly, M Coscolla, M Hanekom, Markers European Concerted Action on New Generation Genetic, MM Gutacker, MW Borgdorff, N Lari, O Neyrolles, P Supply, P Supply, R Staden, RX Liu, S Borrell, S Borrell, S Feuerriegel, S Gagneux, S Gagneux, S Narayanan, S Niemann, S Sreevatsan, Sebastien Gagneux, Sonia Borrell, T Iwamoto, T Lillebaek, TC Victor, TN Buu, TN Buu, UB Singh, US Rajapaksa, Y Hu, Y Pang, YJ Sun, YL Guo   +67 more
core   +3 more sources

Class IIa HDACs forced degradation allows resensitization of oxaliplatin‐resistant FBXW7‐mutated colorectal cancer

Molecular Oncology, EarlyView.
HDAC4 is degraded by the E3 ligase FBXW7. In colorectal cancer, FBXW7 mutations prevent HDAC4 degradation, leading to oxaliplatin resistance. Forced degradation of HDAC4 using a PROTAC compound restores drug sensitivity by resetting the super‐enhancer landscape, reprogramming the epigenetic state of FBXW7‐mutated cells to resemble oxaliplatin ...
Vanessa Tolotto, Nicolò Gualandi, Ylenia Cortolezzis, Raffaella Picco, Monica Colitti, Francesca D'Este, Mariachiara Gani, Wayne W. Hancock, Giovanni Terrosu, Cristina Degrassi, Francesca Agostini, Claudio Brancolini, Luigi E. Xodo, Eros Di Giorgio   +13 more
wiley   +1 more source

The orphan receptor GPR35 contributes to angiotensin II–induced hypertension and cardiac dysfunction in mice [PDF]

, 2018
BACKGROUND: The orphan receptor G protein–coupled receptor 35 (GPR35) has been associated with a range of diseases, including cancer, inflammatory bowel disease, diabetes, hypertension, and heart failure.
Divorty, Nina, Graham, Delyth, Milligan, Graeme, Nicklin, Stuart A.   +3 more
core   +1 more source

Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU

BMJ Open, 2019
Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).Design and setting Identification of all cancer drugs (initial or ...
Aaron S Kesselheim, Kerstin Noëlle Vokinger   +1 more
doaj   +1 more source

ATF4‐mediated stress response as a therapeutic vulnerability in chordoma

Molecular Oncology, EarlyView.
We screened 5 chordoma cell lines against 100+ inhibitors of epigenetic and metabolic pathways and kinases and identified halofuginone, a tRNA synthetase inhibitor. Mechanistically halofuginone induces an integrated stress response, with eIF2alpha phosphorylation, activation of ATF4 and its target genes CHOP, ASNS, INHBE leading to cell death ...
Lucia Cottone, James Dunford, Eleanor Calcutt, Vicki Gamble, Filiz Senbabaoglu Aksu, Lorena Ligammari, Georgina Wherry, Giorgia Gaeta, John C. Christianson, Adrienne M. Flanagan, Udo Oppermann, Adam P. Cribbs   +11 more
wiley   +1 more source

Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]

, 2006
Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and ...
Claxton, K., McCabe, C., Raftery, J., Tsuchiya, A.   +3 more
core  

The photoswitchable cannabinoid azo‐HU308 enables optical control of Ca2+ dynamics in INS‐1 β‐cells via off‐target effects on TRPC channels

FEBS Open Bio, EarlyView.
Light activation of the photoswitchable cannabinoid ligand azo‐HU308 triggers Ca2+ influx in pancreatic β‐cells through TRPC channels, independent of CB2 cannabinoid receptors. This reveals a non‐GPCR pathway for cannabinoid modulation of β‐cell Ca2+ dynamics and establishes azo‐HU308 as an optical tool to study cannabinoid signaling through TRP ...
Alexander E. G. Viray, James A. Frank
wiley   +1 more source

Investigating the landscape of US orphan product approvals

Orphanet Journal of Rare Diseases, 2018
Background The Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan designations that have been granted or ...
Kathleen L. Miller, Michael Lanthier
doaj   +1 more source

Orphan drugs in Italy: availability and time-to-access at regional level

Global & Regional Health Technology Assessment, 2023
Introduction: There are an estimated 26 million rare disease patients in Europe, about 2 million in Italy. Access to orphan drugs has been evaluated nationally and internationally, and delays have been observed due to evaluation of price and therapeutic ...
Maria Lucia Marino, Eva Alessi, Aurora Di Filippo, Barbara Polistena, Francesco Macchia, Federico Spandonaro, Francesco Trotta   +6 more
doaj   +1 more source

Clinically Relevant Outcome Measures in Women With Adrenoleukodystrophy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Adrenoleukodystrophy is a rare inherited peroxisomal disease caused by pathogenic variants in the ABCD1 gene located on the X chromosome. Although the most severe central nervous system and adrenal complications typically affect only men with adrenoleukodystrophy, the majority of women develop myeloneuropathy symptoms in adulthood.
Chenwei Yan, Elizabeth I. Pierpont, Amena S. Fine, Reena V. Kartha   +3 more
wiley   +1 more source