Results 281 to 290 of about 3,868,024 (353)

Multicenter Single‐Blind Randomized Controlled Trial of the Romiplostim Biosimilar

eJHaem, Volume 6, Issue 4, August 2025.
Abstract Background This was a randomized multicenter single‐blinded active‐controlled equivalence Phase III study evaluating the efficacy and safety of the biosimilar of romiplostim (GP40141) compared to the reference drug Nplate in patients with persistent or chronic immune thrombocytopenia (ITP).
Anait L. Melikyan, Ekaterina A. Protsenko, Galina N. Salogub, Bulat A. Bakirov, Igor L. Davydkin, Vladislav V. Kovalik, Mariia L. Gefen, Yuliia D. Matvienko, Valeria B. Saparova, Alexander L. Khokhlov, Igor E. Makarenko, Roman V. Drai   +11 more
wiley   +1 more source

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. [PDF]

Pharmaceuticals (Basel)
Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A.   +7 more
europepmc   +1 more source

An Early Examination of Access to Select Orphan Drugs Treating Rare Diseases in Health Insurance Exchange Plans

, 2014
Sandy W. Robinson, Kelly Brantley, Christine Liow, J. Russell Teagarden   +3 more
openalex   +2 more sources

Mangiferin Against Respiratory Diseases: Pharmacological Targets and Prospects

Pharmacology Research &Perspectives, Volume 13, Issue 4, August 2025.
ABSTRACT Respiratory diseases are associated with high mortality worldwide. Respiratory infections can lead to the emergence of chronic respiratory diseases. Scientists are constantly striving to identify new therapies with reduced side effects.
Kazi Ahsan Ahmed, Nusrat Afrin, Popy Ghosh, Irin Amin Heya, Sajidur Rahman Akash, Akhi Moni, Mohammad Nazrul Islam, Md. Golzar Hossain, Alessandra Sinopoli, Md Abdul Hannan, Md Jamal Uddin   +10 more
wiley   +1 more source

Genome Variation in Alcohol Use Disorder by Whole‐Exome Sequencing

Addiction Biology, Volume 30, Issue 8, August 2025.
Whole‐exome sequencing of 83 alcohol use disorder patients identified 106,525 SNVs and 19,826 InDels, revealing six genes (CNTNAP3, ZNF683, ALDH2, CCHCR1, ZNF45, ESRRA) with deleterious mutations through comprehensive bioinformatics analysis. These novel genetic variants represent potential targets for precision medicine in alcohol use disorder ...
Lei Liu, Bo Zhang, Yong Dong, Li Ping Liu, Jing Ying Wang, Jun Liu, Guang Yu Zhou, Chuan Yi Kang, Xiaorui Hu, Chang Cheng, Na Zhao, Jia Lu, Huaizhi Wang, Jian Hu, Xiaohong Wang   +14 more
wiley   +1 more source

Efficacy of IDOR‐1117‐2520, a novel, orally available CCR6 antagonist in preclinical models of skin dermatitis

British Journal of Pharmacology, Volume 182, Issue 15, Page 3452-3475, August 2025.
Background and Purpose The chemokine receptor CCR6 guides pathogenic T17 cells, implicated in autoimmune diseases including psoriasis, to sites of inflammation via the chemokine CCL20. Therefor, pharmacological inhibition of CCR6+ immune cell migration provides a novel therapeutic approach.
Paulina Kulig, Pijus Brazauskas, Madeleine Suffiotti, Emilie Raoult, Ulrike Babilonski, Bérengère Renault, Ursula Grieder, Enrico Vezzali, Peter Blattmann, Marianne M. Martinic, Mark J. Murphy   +10 more
wiley   +1 more source

Dimerisation of the VIP receptor VIPR2 is essential to its binding VIP and Gαi proteins, and to its functions in breast cancer cells

British Journal of Pharmacology, Volume 182, Issue 15, Page 3612-3627, August 2025.
Abstract Background and Purpose Several G protein‐coupled receptors (GPCRs) are known to homodimerise. Dimeric GPCRs may have different properties from their monomers, but the molecular basis and functional significance of GPCR dimerisation remain largely unknown.
Satoshi Asano, Kairi Ozasa, Teru Uehara, Rei Yokoyama, Takanobu Nakazawa, Souichi Yanamoto, Yukio Ago   +6 more
wiley   +1 more source

Artemisinins in autoimmune diseases: effects and mechanisms in systemic lupus erythematosus and rheumatoid arthritis

British Journal of Pharmacology, Volume 182, Issue 15, Page 3411-3427, August 2025.
Abstract Autoimmune diseases such as systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA) result from immune self‐tolerance loss, causing tissue damage. Standard treatments targeting late‐stage inflammation often have serious side effects. Artemisinin, known for its antimalarial properties, shows potential immunomodulatory effects.
Fanchao Zhou, Guoming Li, Ruixiang Tan, Guodong Wu, Changsheng Deng   +4 more
wiley   +1 more source

Orphan Drug Designation to Aldoxorubicin for Glioblastoma, Small Cell Lung Cancer, & Ovarian Cancer

, 2014
NULL AUTHOR_ID
openalex   +1 more source