The orphan drug dichloroacetate reduces amyloid beta-peptide production whilst promoting non-amyloidogenic proteolysis of the amyloid precursor protein [PDF]
, 2021 Abstract The amyloid cascade hypothesis proposes that excessive accumulation of amyloid beta-peptides is the initiating event in Alzheimer’s disease. These neurotoxic peptides are generated from the amyloid precursor protein via sequential cleavage by β- and γ-secretases in the ‘amyloidogenic’ proteolytic pathway ...
Edward T. Parkin +2 more
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Review of: "The orphan drug dichloroacetate reduces amyloid beta-peptide production whilst promoting non-amyloidogenic proteolysis of the amyloid precursor protein" [PDF]
, 2021 Darrell D. Mousseau
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The drug development pipeline for glioblastoma—A cross sectional assessment of the FDA Orphan Drug Product designation database [PDF]
PLOS ONE, 2021 Background Glioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the molecular makeup of this disease has been increasing rapidly ...
Pascal Johann, Dominic Lenz, Markus Ries
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Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest. [PDF]
Clin Transl SciABSTRACT Rare diseases affect a small population of patients, resulting in low incentives for developing orphan drug products (ODPs). The United States Congress passed the Orphan Drug Act of 1983 to incentivize pharmaceutical manufacturers to develop drugs to treat rare diseases.
Hopefl R +6 more
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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
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Orphan drugs and the NHS: Should we value rarity [PDF]
, 2005 Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients.
Claxton, K., McCabe, C., Tsuchiya, A.
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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
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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
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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
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Nuclear receptors in vascular biology [PDF]
, 2015 Nuclear receptors sense a wide range of steroids and hormones (estrogens, progesterone, androgens, glucocorticoid, and mineralocorticoid), vitamins (A and D), lipid metabolites, carbohydrates, and xenobiotics.
A Chawla +56 more
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