Results 171 to 180 of about 160,917 (252)

Management of T‐cell malignancies: Bench‐to‐bedside targeting of epigenetic biology

CA: A Cancer Journal for Clinicians, Volume 75, Issue 4, Page 282-307, July/August 2025.
Abstract The peripheral T‐cell lymphomas (PTCL) are the only disease for which four histone deacetylase (HDAC) inhibitors have been approved globally as single agents. Although it is not clear why the PTCL exhibit such a vulnerability to these drugs, understanding the biological basis for this activity is essential.
Ariana Sabzevari, Johnson Ung, Jeffrey W. Craig, Kallesh D. Jayappa, Ipsita Pal, David J. Feith, Thomas P. Loughran Jr, Owen A. O’Connor   +7 more
wiley   +1 more source

[Rare diseases: the role of internal medicine]. [PDF]

Inn Med (Heidelb)
Sauerbruch T, Aretz S, Hebestreit H, Kaemmerer H, Nährlich L, Siegmund B, Ertl G, Deutsche Gesellschaft für Innere Medizin (DGIM).   +7 more
europepmc   +1 more source

Nuclear Receptor ERRγ Protects Against Cardiac Ischemic Injury by Suppressing GBP5‐Mediated Myocardial Inflammation

The FASEB Journal, Volume 39, Issue 14, 31 July 2025.
Cardiomyocyte‐specific overexpression of ERRγ after myocardial infarction improves cardiac function by suppressing the expression of its pro‐inflammatory downstream target GBP5, thereby inhibiting inflammasome assembly, alleviating myocardial inflammation, and pyroptosis.
Junhao Qiu, Qianji Che, Yichao Zhang, Mu Chen, Zhixing Wei, Yangjinming Bai, Tingting Zhao, Ji Yan, Zhengyang Wu, Zhentao Fei, Yuepeng Wang, Qian Wang, Yi‐Gang Li   +12 more
wiley   +1 more source

Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023. [PDF]

Front Pharmacol
Nie R, Zhao Z, Zhang Y, Xu B, Zhang W.
europepmc   +1 more source

Novel Para‐Phenylenediamine‐Based Derivatives as Receptor Tyrosine Kinase‐like Orphan Receptor 1 (ROR1) Inhibitors: An In Vitro Preliminary Characterization

ChemMedChem, Volume 20, Issue 14, July 18, 2025.
ROR1 represents a promising target for the development of novel antiproliferative compounds, giving its high expression in different cancer cell lines. The present study describes the workflow leading to the design, synthesis, and characterization of a series of para‐phenylenediamine‐based compounds able to interact with the target kinase, inhibiting ...
Gerardina Smaldone, Maria Rosaria Miranda, Francesca Di Matteo, Valeria Napolitano, Michela Aliberti, Simona Musella, Veronica Di Sarno, Gianluigi Lauro, Giuseppe Bifulco, Giacomo Pepe, Giovanna Aquino, Mario Felice Tecce, Isabel Maria Gomez‐Monterrey, Pietro Campiglia, Carmine Ostacolo, Alessia Bertamino, Vincenzo Vestuto, Tania Ciaglia   +17 more
wiley   +1 more source

Application of observational research methods to real-world studies for rare disease drugs: A scoping review protocol. [PDF]

PLoS One
Patel YP, Ghaddar L, Lin Y, Karim N, Chan K, Dupuis L, Tadrous M.   +6 more
europepmc   +1 more source

Orphan Nuclear Receptors TR2 and TR4 in Erythropoiesis: From Mechanisms to Therapies. [PDF]

Biomolecules
Liu Y, Yang H, Ren M, Yu Q, Xu Q, Fu X.
europepmc   +1 more source

Evolving Research and Development Landscape for Rare Diseases: Growing Concerns Over Orphan Drug Lag in Japan. [PDF]

Clin Pharmacol Ther
Enya K, Kageyama I, Kobayashi Y, Lim Y, Sengoku S, Kodama K.   +5 more
europepmc   +1 more source

Guiding the willingness to pay for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome using a cost-based-plus pricing framework

Manders EA, Keestra S, Bannenberg WJ, de Visser SJ, Hollak CE.   +4 more
europepmc   +1 more source

Orphan nuclear receptor transcription factors as drug targets. [PDF]

Transcription
Safe S, Oany AR, Tsui WN, Lee M, Srivastava V, Upadhyay S, Hailemariam A, Farkas E, Kakwan S, Kearns C, Sivaram G.   +10 more
europepmc   +1 more source