Results 81 to 90 of about 3,868,024 (353)
Functional profiles of orphan membrane transporters in the life cycle of the malaria parasite [PDF]
Assigning function to orphan membrane transport proteins and prioritizing candidates for detailed biochemical characterization remain fundamental challenges and are particularly important for medically relevant pathogens, such as malaria parasites.
Kenthirapalan, Sanketha+3 more
core +4 more sources
This review explores the potential of mechanochemistry in the late‐stage modification of active pharmaceutical ingredients (APIs), offering a comprehensive analysis of methods designed to transform structurally complex molecular scaffolds. By examining the scope, efficiency, and mechanistic aspects of these approaches, the review highlights protocols ...
Johanna Templ, Lars Borchardt
wiley +1 more source
Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?
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
Orphan Drug Pricing and Costs: A Case Study of Kalydeco and Orkambi
Background: A common narrative is that high prices are necessary for “orphan drugs” because of the fewer patients. In the context of state health insurance systems, the high prices create significant challenges because of limited budgets.
A. Hollis
semanticscholar +1 more source
Generating health technology assessment evidence for rare diseases [PDF]
Objectives: Rare diseases are often heterogeneous in their progression and response to treatment, with only a small population for study. This provides challenges for evidence generation to support HTA, so novel research methods are required.
Facey, Karen+6 more
core +1 more source
Objective Autoimmune diseases, such as systemic lupus erythematosus (SLE), are associated with pulmonary arterial hypertension (PAH), a condition that can lead to heart failure. However, whether T cells also contribute to the occurrence of PAH in SLE has not been clarified.
Shuliang Jing+9 more
wiley +1 more source
Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]
While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core +1 more source
Estrogen-related receptor α (ERRα) : a novel target in type 2 diabetes [PDF]
Recent studies have shown that reduced mitochondrial content and function in skeletal muscle are common features of type 2 diabetes. Here, we review the molecular mechanisms involved in the regulation of mitochondrial genes in skeletal muscle, focusing ...
Handschin, C., Mootha, V. K.
core +1 more source
Mucosal‐Associated Invariant T Cells in Rheumatic Diseases
Mucosal‐associated invariant T (MAIT) cells are innate‐like T cells defined by their semi‐invariant T cell receptor and restriction by the major histocompatibility complex class I–related molecule (MR1). These cells are primarily activated by microbial‐derived metabolites presented by MR1 or by cytokines.
Manon Lesturgie‐Talarek+7 more
wiley +1 more source
Pricing and reimbursement of orphan drugs: the need for more transparency
Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients.
Simoens Steven
doaj +1 more source