Results 161 to 170 of about 22,997 (249)

Lack of Differences in the Pharmacokinetics of Therapeutic Monoclonal Antibodies Between Japanese and Non‐Japanese Individuals

The Journal of Clinical Pharmacology, Volume 66, Issue 8, August 2026.
Abstract Evaluation of the pharmacokinetics (PK) of new drugs in Japanese individuals is regularly requested by the Japanese regulatory agency prior to participation in global clinical trials or for bridging approaches for regulatory approval. However, the need for PK assessments in the Japanese population may be less compelling for monoclonal ...
Paridhi Gupta, Ashish Sharma, Bernd Meibohm   +2 more
wiley   +1 more source

Assessing rare disease understanding: a novel disease readiness level framework. [PDF]

Orphanet J Rare Dis
Kitahara K, Kano S.
europepmc   +1 more source

Repercussions of Diagnostic Delay in Rare Diseases

Journal of Genetic Counseling, Volume 35, Issue 4, August 2026.
ABSTRACT Rare diseases (RDs) are often subject to diagnostic delays due to their low prevalence, clinical variability, and limited professional awareness. This scoping review aimed to map the literature on these delays, examining their clinical, emotional, and socioeconomic consequences.
Luisa Rezende Batista, Luiza Fernandes Valente, Fernando Spina, Davi Said Gonçalves Celso, Israel Gomy, Christiane Mariotini‐Moura   +5 more
wiley   +1 more source

Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study. [PDF]

Int J Health Policy Manag
Lexchin J.
europepmc   +1 more source

Tumor‐Responsive PLGA‐SS‐HA Nanoparticles for Zinc Complex Delivery Suppress Prostate Cancer via TR3/FTH1 Pathway

Macromolecular Materials and Engineering, Volume 311, Issue 8, August 2026.
A tumor‐responsive PLGA‐SS‐HA nanoplatform enables glutathione‐triggered delivery of zinc complexes to prostate cancer cells. Enhanced intracellular zinc accumulation induces oxidative stress, mitochondrial dysfunction, and apoptosis through activation of the TR3/FTH1 signaling pathway.
Ding Ma, Sheng Ge, Ruimin Ren, Yongqiang Fu   +3 more
wiley   +1 more source

The EMA as a potential driver of competitiveness of orphan medicines in Europe: a focus on regulatory incentives and funding opportunities. [PDF]

Orphanet J Rare Dis
Gomez-Ferreria MA, Garcia-Muñoz V, Zwiers A.   +2 more
europepmc   +1 more source

Medications That Regulate Pregnane X Receptor: A Systematic Review of Current Evidence

Pharmacology Research &Perspectives, Volume 14, Issue 4, August 2026.
PRISMA 2020 flow diagram of study selection for this systematic review of medications that regulate the pregnane X receptor (PXR), from 12 236 records screened to 101 included studies. ABSTRACT The pregnane X receptor (PXR) gene encodes a ligand‐activated protein involved with the metabolism and excretion of drugs, toxins, and other xenobiotics.
Petra Czarniak, Rachael Moorin, Jeff Hughes, Karl Gruber, David Youens   +4 more
wiley   +1 more source

Increasing access to CAR-T therapy: a case study of an academic hospital's alternative innovation model. [PDF]

Orphanet J Rare Dis
Ruiz AA, Shinabargar E, Slovenski I, Large K, Vieira M, Moon S.   +5 more
europepmc   +1 more source

The Relationship Between (Endo)cannabinoids, the Microbiome and Melanoma

Pharmacology Research &Perspectives, Volume 14, Issue 4, August 2026.
CB1 and CB2 receptors are widely distributed in both normal and cancerous cells. Cannabinoids have a positive impact on reducing tumor growth in mice. A positive relationship exists between CB2 receptors and survival in skin melanoma. However, there is a negative impact on non‐small cell lung cancer and colon cancer, whereas a higher association of CB1
Farideh A. Javid, Marin Golčić, Anja Harej Hrkać, Andrej Belančić   +3 more
wiley   +1 more source