Results 141 to 150 of about 22,884 (253)

GPR3 Ligands Discovered through Combined Virtual and Conformational Biosensor-Based Screening. [PDF]

J Am Chem Soc
Schihada H, Shahraki A, Turku-Metsänen A, Rath M, Wirth L, Nemec K, Tselepli H, Heitzer L, Vallaster B, Fadel M, Schulte G, Hilger D, Pockes S, Lohse MJ, Kolb P.   +14 more
europepmc   +1 more source

A Systematic Review of Adverse Childhood Experiences and Epigenetic Age Acceleration in Later Adult Life Measured With Second and Third‐Generation Epigenetic Clocks

American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, Volume 201, Issue 4, Page 270-290, June 2026.
ABSTRACT We examined the relationship between adverse childhood experiences (ACEs) and epigenetic age acceleration (EAA) in adulthood as measured by second and third generation epigenetic clocks by performing a systematic review of the literature. The electronic databases MEDLINE and EMBASE were searched on 17 July 2023.
Matthew Green, Laura Horsfall, Azam Saied   +2 more
wiley   +1 more source

Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China. [PDF]

Regen Ther
Shi J, Hu H, Ung COL.
europepmc   +1 more source

Silymarin and Its Isolated Flavanonol Lignans Are Liver X Receptor Antagonists

Archiv der Pharmazie, Volume 359, Issue 6, June 2026.
Despite its long tradition in the treatment of liver‐related ailments, the precise mode of action of the milk thistle fruit extract remains vague. Here we identified liver X receptor as a molecular target of silymarin, a mixture of bioactive compounds from milk thistle extract, and its major flavanonol lignan components, which account for their anti ...
Nils C. Bandomir, Astrid Kaiser, Felix F. Lillich, Pascal Heitel   +3 more
wiley   +1 more source

Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]

Clin Transl Sci
Fletcher EP, Kim C, Mei H, AbuAsal B, Samuels S, Madabushi R, Ramamoorthy A.   +6 more
europepmc   +1 more source

Evaluation and Valuation of the Price of Expensive Medicinal Products: Application of the Discounted Cash Flow to Orphan Drugs

International Journal of Rare Diseases & Disorders, 2018
Nuijten Mark, Fugel Hans Joerg, Vis Jan
openaire   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, Volume 92, Issue 6, Page 1552-1566, June 2026.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion. [PDF]

Health Expect
Frost J, Widening Engagement Patient Advisory Group, Mandizha J, van den Dungen C, Asare L, Pope C.   +5 more
europepmc   +1 more source

Mu Opioid Receptor mRNA and Protein Localization Across the Rat and Mouse Habenula

Journal of Comparative Neurology, Volume 534, Issue 6, June 2026.
Mu opioid receptor (MOR) mRNA was detected with RNAscope in situ hybridization for Oprm1 and protein was detected with immunocytochemistry to map the distributions across the anterior‐posterior extent of the rat and mouse habenula. Expression distribution was also characterized in a transgenic reporter mouse line (Oprm1‐Venus/Venus) as an additional ...
Aashka K. Popat, Rhiana C. Simon, Beatriz B. Aoyama, Ahana Wokhlu, Aliza T. Ehrlich, Corey C. Harwell, Elyssa B. Margolis   +6 more
wiley   +1 more source

Consequences of Canada's Drug Agency Reimbursement Recommendations for New Medicines and Pan-Canadian Pharmaceutical Alliance Price Negotiations on Patient Access. [PDF]

Clinicoecon Outcomes Res
Rawson NSB.
europepmc   +1 more source