Results 11 to 20 of about 8,062,145 (231)

New role of extemporaneous manufacturing in regulating drug products access onto the market

open access: yesФармация и фармакология (Пятигорск), 2023
The aim of the work was to study the legal aspects of the legislative regulation for manufacturing medicines in a pharmacy organization.Materials and methods.
A. V. Alekhin   +5 more
doaj   +1 more source

Small heterodimer partner-targeting therapy inhibits systemic inflammatory responses through mitochondrial uncoupling protein 2. [PDF]

open access: yesPLoS ONE, 2013
The orphan nuclear receptor, small heterodimer partner (SHP), appears to play a negative regulatory role in innate immune signaling. Emerging evidence warrants further study on the therapeutic targeting of SHP to suppress excessive and deleterious ...
Chul-Su Yang   +8 more
doaj   +1 more source

Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?

open access: yesVoices in Bioethics, 2019
The mainstream media and general public have expressed frustration in recent months with the confluence of capitalism and the pharmaceutical research and development enterprise.
Michael Menconi
doaj   +1 more source

Metformin-Inducible Small Heterodimer Partner Interacting Leucine Zipper Protein Ameliorates Intestinal Inflammation

open access: yesFrontiers in Immunology, 2021
Small heterodimer partner interacting leucine zipper protein (SMILE) is an orphan nuclear receptor and a member of the bZIP family of proteins. We investigated the mechanism by which SMILE suppressed the development of inflammatory bowel disease (IBD ...
SeungCheon Yang   +16 more
doaj   +1 more source

The Vital Role Played by Deferiprone in the Transition of Thalassaemia from a Fatal to a Chronic Disease and Challenges in Its Repurposing for Use in Non-Iron-Loaded Diseases

open access: yesPharmaceuticals, 2023
The iron chelating orphan drug deferiprone (L1), discovered over 40 years ago, has been used daily by patients across the world at high doses (75–100 mg/kg) for more than 30 years with no serious toxicity.
George J. Kontoghiorghes
doaj   +1 more source

Product development and quality of pharmacy compounded chenodeoxycholic acid capsules for Dutch cerebrotendinous xanthomatosis patients

open access: yesFrontiers in Pharmacology, 2023
Introduction: In 2017 the drug chenodeoxycholic acid (CDCA) became unavailable to Dutch patients with the rare inborn error of metabolism cerebrotendinous xanthomatosis (CTX).
Natalja Bouwhuis   +4 more
doaj   +1 more source

Production of chain-extended cinnamoyl compounds by overexpressing two adjacent cluster-situated LuxR regulators in Streptomyces globisporus C-1027

open access: yesFrontiers in Microbiology, 2022
Natural products from microorganisms are important sources for drug discovery. With the development of high-throughput sequencing technology and bioinformatics, a large amount of uncharacterized biosynthetic gene clusters (BGCs) in microorganisms have ...
Xingxing Li   +10 more
doaj   +1 more source

Cryo‐EM structure of orphan G protein‐coupled receptor GPR21

open access: yesMedComm, 2023
GPR21 belongs to class A orphan G protein‐coupled receptor (GPCR). The endogenous ligands for human GPR21 remain unidentified. GPR21 expression is associated with developing type 2 diabetes (T2DM), a multifactorial metabolic disease caused by pancreatic ...
Thian‐Sze Wong   +8 more
doaj   +1 more source

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

open access: yesOrphanet Journal of Rare Diseases, 2018
Background To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing ...
Caridad Pontes   +13 more
doaj   +1 more source

Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC

open access: yesHepatology, EarlyView., 2022
Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu   +11 more
wiley   +1 more source

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