Results 11 to 20 of about 68,610 (290)
Orphan products: an emerging trend in drug approvals [PDF]
Nature Reviews Drug Discovery, 2010 Timothy, Coté +4 more
openaire +4 more sources
Frontiers 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
Gene and Cell Therapies Overview Under the Light of Health Economics [PDF]
Health Economics and Management Review, 2022 With the increase in drug development studies for rare diseases, gene therapies have recently come to the fore more frequently. In addition to orphan drugs used in the treatment of rare diseases, advanced therapy medicinal products have been developed ...
Ekin Begum Karahan, Guvenc Kockaya
doaj +1 more source
Small heterodimer partner-targeting therapy inhibits systemic inflammatory responses through mitochondrial uncoupling protein 2. [PDF]
PLoS 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
Physiopathological Implications of 7TM Receptors [PDF]
, 2010 Seven-transmembrane (7TM) receptors are one of the most important proteins involved in perception of extracellular stimuli and regulation of variety of intracellular signaling pathways.
Cygankiewicz, Adam
core +2 more sources
Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?
Voices 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
Pharmaceuticals, 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
Pharmaceutical compounding of orphan active ingredients in Belgium : how community and hospital pharmacists can address the needs of patients with rare diseases [PDF]
, 2019 Background: Pharmaceutical compounding of orphan active ingredients can offer cost-effective treatment to patients when no other drug product is available for a rare disease or during periods of drug product shortages.
Boussery, Koen +7 more
core +2 more sources
Frontiers 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
Frontiers 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