An integrative neuropharmacological review of Huntington's disease challenges and the role of novel formulations in addressing pharmacological‒pharmaceutical limitations. [PDF]
Senousy MA +7 more
europepmc +1 more source
Opportunities and challenges in protein structure prediction
Abstract Deep learning methods, particularly exemplified by AlphaFold2, have revolutionized the field of protein structure prediction—an achievement recognized by the 2024 Nobel Prize in Chemistry awarded to its core developers. Despite this remarkable achievement, the broader protein folding problem is far from solved. Key challenges—each representing
Wenkai Wang +3 more
wiley +1 more source
Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands. [PDF]
Versteeg JW +5 more
europepmc +1 more source
When Public Health Becomes the Weapon: Current and Prospective Consequences of the Genocide in Gaza
ABSTRACT Catastrophic humanitarian conditions during the 2023–2025 genocide in Gaza have caused a public health crisis of exceptional magnitude. This article summarizes key short‐ and long‐term health consequences for Gaza's civilian population and outlines priorities for recovery.
Therese Alexandra Evald +19 more
wiley +1 more source
Advances in Cell and Gene Therapy for Rare Disease Treatment. [PDF]
Baek HJ, Nam Y, Rim YA, Ju JH.
europepmc +1 more source
The molecular mechanisms of RORA targeting PRNP activate oxidative injury‐induced cellular senescence and apoptosis of lens epithelial cells. Current research confirms that the transcription factor RORA targets PRNP, exacerbating cellular senescence and apoptosis of LECs (Figure 7).
Yue Zou +11 more
wiley +1 more source
Pregnancies in women with rare diseases: Selected maternal and perinatal outcomes
Pregnancies in women with rare diseases carry substantial disease‐related (23.2%) and pregnancy‐specific (25.1%) risks. Complication rates drop markedly when conditions are stable before conception. Abstract Introduction Rare diseases (RD) are characterized by chronicity and may be associated with reduced life expectancy and quality of life.
Philipp Kosian +6 more
wiley +1 more source
How to evaluate rare disease policy effectiveness based on policy modeling consistency (PMC) index model: A quantitative assessment of policy implementation in China. [PDF]
Zhao Y +6 more
europepmc +1 more source
Summary Real‐world data on treatment response after switching between thrombopoietin receptor agonists are limited for patients with immune thrombocytopenia (ITP). The objective of this study is to describe treatment patterns and outcomes in patients with primary ITP who switched from eltrombopag (ELT) or romiplostim (ROMI) to avatrombopag (AVA).
Caroline Piatek +8 more
wiley +1 more source
Background and Purpose Intestinal barrier dysfunction caused by mitochondrial stress, oxidative damage and apoptosis, are hallmarks of dysbiosis‐associated gastrointestinal (GI) disorders. Staurosporine causes downstream features of dysbiosis‐induced epithelial damage.
Fatima Maqoud +9 more
wiley +1 more source

