Results 171 to 180 of about 68,732 (285)

Federal 340B Program Payment Scheme for Drugs Designated As Orphan Products: Congressional Clarification Needed to Close the Government-Industry Revolving Door [PDF]

Journal of Clinical Oncology, 2016
Yang, Y. Tony, Chen, Brian, Bennett, Charles L.   +2 more
openaire   +2 more sources

Impacts of exercise, renin–angiotensin system modulation or both on skeletal muscle circadian gene expression

Experimental Physiology, EarlyView.
Abstract Ageing negatively affects quality of life and healthspan, and interventions are needed to slow this progressive decline. Previously, we have demonstrated the potential functional benefits of combining a genetically modified probiotic (GMP) targeting the non‐canonical arm of the renin–angiotensin system (RAS) with exercise training.
Emily L. Zumbro, Liliana C. Baptista, Taylor Taylor, Abbi R. Hernandez, Anisha Banerjee, Yi Sun, YouFeng Yang, Qiuhong Li, Thomas W. Buford   +8 more
wiley   +1 more source

Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands. [PDF]

Int J Technol Assess Health Care
Versteeg JW, Stam N, Mantel-Teeuwisse AK, Timmers L, Goettsch W, Leopold C.   +5 more
europepmc   +1 more source

Hypoxia and the cytoskeleton

The Journal of Physiology, EarlyView.
Abstract figure legend Schematic outlining the activation of hypoxia‐sensitive pathways, the influence of hypoxia and associated pathways on the cytoskeleton, and the impact of these on disease progression. Abstract A highly‐regulated and dynamic cytoskeleton is vital for functional cellular physiology and the maintenance of homeostasis.
Darragh Flood, Cormac T. Taylor
wiley   +1 more source

Advances in Cell and Gene Therapy for Rare Disease Treatment. [PDF]

Int J Stem Cells
Baek HJ, Nam Y, Rim YA, Ju JH.
europepmc   +1 more source

In Vitro Metabolism and Analytical Characterization of SLU‐PP‐332 and SLU‐PP‐915: Novel Pan‐ERR Agonists With Doping Potential

Rapid Communications in Mass Spectrometry, Volume 40, Issue 8, 30 April 2026.
ABSTRACT Rationale Estrogen‐related receptor (ERR) agonists such as the drug candidates SLU‐PP‐332 and SLU‐PP‐915 are currently being investigated as exercise mimetics, given their ability to trigger human physiological processes similar to those initiated by actual physical activity.
Tristan Möller, Oliver Krug, Mario Thevis   +2 more
wiley   +1 more source

Strategic Drivers Behind Early Withdrawal of Orphan Designations in the EU: A Retrospective Analysis (2000-2024). [PDF]

Ther Innov Regul Sci
Bouwman L, Castanheira M, Siotis G.
europepmc   +1 more source

Development and Validation of an LC–MS/MS Method for the Quantitative Estimation of Bexicaserin in Human Cerebrospinal Fluid

Biomedical Chromatography, Volume 40, Issue 3, March 2026.
ABSTRACT Bexicaserin is an investigational molecule that is a highly selective superagonist of the 5‐hydroxytryptamine 2C (5HT2c) serotonin receptor subtype and in Phase 3 clinical studies for the treatment of seizures associated with developmental and epileptic encephalopathies (DEEs).
Raja Reddy Kallem, Rosa Chan, Katie Neal, Maisy Yeager, Katharine Fletcher, Nuggehally R. Srinivas   +5 more
wiley   +1 more source

HTA Evidence in Rare Diseases: Just Rare or Also Special? [PDF]

Pharmacoeconomics
Basu A, Thomas SK, Chapman RH, Spangler J.   +3 more
europepmc   +1 more source

Pharmacogenetic Interventions, Orphan Groups, and Distributive Justice: The Role of Cost-Benefit Analysis [PDF]

, 2002
Rai, Arti K.
core   +1 more source