Results 1 to 10 of about 160,917 (252)

Plant molecular farming: a promising frontier for orphan drug production

open access: closedBiotechnology Letters
Orphan diseases comprise a range of disorders that individually affect a small percentage of people, but collectively impact millions of people worldwide. Patients with this disorder often face significant challenges in diagnosis, treatment, and access to care due to their rare nature and limited understanding and treatment options.
Balamurugan Shanmugaraj   +2 more
semanticscholar   +4 more sources
Some of the next articles are maybe not open access.

Development of Ligands for the Super Conserved Orphan G Protein-Coupled Receptor GPR27 with Improved Efficacy and Potency.

Journal of Medicinal Chemistry, 2023
The orphan G protein-coupled receptor GPR27 appears to play a role in insulin production, secretion, lipid metabolism, neuronal plasticity, and l-lactate homeostasis. However, investigations on the function of GPR27 are impaired by the lack of potent and
Thanigaimalai Pillaiyar   +9 more
semanticscholar   +1 more source

Retinoic acid-related orphan receptor gamma t (RORγt) inverse agonists/antagonists for the treatment of inflammatory diseases – where are we presently?

Expert Opinion on Drug Discovery, 2021
Introduction: The transcription factor retinoic acid-related orphan receptor gamma t (RORγt) has been identified as the master regulator of TH17 cell differentiation and IL-17/22 production and is therefore an attractive target for the treatment of ...
C. Gege
semanticscholar   +1 more source

ORPHAN DRUG POLICY

Innovare Journal of Life Sciences, 2020
Pharmaceutical industry is crucial for mankind and contributes in some way in socio economic development of the society through jobs, community welfare, and supply chains.
R. Goel
semanticscholar   +1 more source

Valhidepsin Lipopeptides from Chromobacterium vaccinii: Structures, Biosynthesis, and Coregulation with FR900359 Production.

Journal of Natural Products, 2023
Microbial secondary metabolites continue to provide a valuable source of both chemical matter and inspiration for drug discovery in a broad range of therapeutic areas.
D. Pistorius   +10 more
semanticscholar   +1 more source

Dutch hospital resumes production of rare drug after sourcing purer ingredients

British medical journal, 2020
A university hospital in the Netherlands has resumed cost price production of orphan medicine chenodeoxycholic acid (CDCA), a primary bile acid used to treat the rare hereditary metabolic disorder cerebrotendinous xanthomatosis (CTX). The pharmaceutical
T. Sheldon
semanticscholar   +1 more source

A Drug with Lipophilicity-Dependent Potency Can Be Metabolically Stable: Discovery of a Potent and Selective Retinoic Acid Receptor-Related Orphan Receptor C2 (RORC2) Inverse Agonist as an Orally Bioavailable Anti-Inflammatory Agent.

Journal of Medicinal Chemistry, 2018
IL-17 drives an amplification mechanism in inflammatory diseases such as psoriasis, psoriatic arthritis, and ankylosing spondylitis. The production of IL-17 depends on the activity of RORC2 in immune cells, which suggests that RORC2 inverse agonists are ...
Yibing Wang, Hong Liu
semanticscholar   +1 more source

Feasibility Study for Bedside Production of Recombinant Human Acid α-Glucosidase: Technical and Financial Considerations.

Current Pharmaceutical Biotechnology, 2020
The high cost of orphan drugs limits their access by many patients, especially in low- and middle-income countries. Many orphan drugs are off-patent without alternative generic or biosimilar versions available.
Mohammed H. Aldosari   +5 more
semanticscholar   +1 more source

Viltolarsen: a treatment option for Duchenne muscular dystrophy patients who are amenable to exon 53 skipping therapy

Expert Review of Neurotherapeutics, 2023
Introduction Duchenne muscular dystrophy (DMD) is a progressive genetic disease leading to muscular weakness. DMD is caused by mutations of the dystrophin gene on the X chromosome that is responsible for production of dystrophin protein.
Eszter Czifrus, Daniel J. Berlau
semanticscholar   +1 more source

Lipid-Dependent Activation of the Orphan G Protein-Coupled Receptor, GPR3

Biochemistry
The class A orphan G protein-coupled receptor (GPCR), GPR3, has been implicated in a variety of conditions, including Alzheimer’s and premature ovarian failure. GPR3 constitutively couples with Gαs, resulting in the production of cAMP in cells.
Isabella C. Russell   +11 more
semanticscholar   +1 more source

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