Results 91 to 100 of about 110,217 (215)
Do investors value the FDA orphan drug designation?
Orphanet Journal of Rare Diseases, 2017 Background The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases.
Kathleen L. Miller
doaj +1 more source
Race, Money and Medicines [PDF]
, 2006 Taking notice of race is both risky and inevitable, in medicine no less than in other endeavors. The literature on race as a classifying tool in clinical research poses this core dilemma: On the one hand, race can be a useful stand-in for unstudied ...
Bloche, Maxwell Gregg
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Advanced NanoBiomed Research, EarlyView.This review highlights how autoimmune diseases arise from intertwined immunological, genetic, and environmental factors, emphasizing gut microbiota dysbiosis as a pivotal driver. It outlines emerging nanotechnology‐based strategies—such as liposomes, hydrogels, and polymeric nanoparticles—that enhance targeted drug delivery, minimize systemic toxicity,
Md. Meraj Ansari +5 more
wiley +1 more source
In silico and in vitro drug screening identifies new therapeutic approaches for Ewing sarcoma. [PDF]
, 2016 The long-term overall survival of Ewing sarcoma (EWS) patients remains poor; less than 30% of patients with metastatic or recurrent disease survive despite aggressive combinations of chemotherapy, radiation and surgery.
Alturkmani, Hani +11 more
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Citation: 'orphan drug' in the IUPAC Compendium of Chemical Terminology, 5th ed.; International Union of Pure and Applied Chemistry; 2025. Online version 5.0.0, 2025. 10.1351/goldbook.12699 • License: The IUPAC Gold Book is licensed under Creative Commons Attribution-ShareAlike CC BY-SA 4.0 International for individual terms.
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Mucosal‐Associated Invariant T Cells in Rheumatic Diseases
Arthritis &Rheumatology, EarlyView.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
Understanding Disease Heterogeneity and Patient Characteristics in Patients with Amyotrophic Lateral Sclerosis (ALS) [PDF]
, 2019 Background: Amytrophic lateral sclerosis (ALS) is a fatal neurologic disease that is projected to double in worldwide incidence in the next 20 years.
Mathew, Veena
core
British Journal of Clinical Pharmacology, EarlyView.Abstract Aims In the context of pharmacology and toxicology education, there is a growing shift toward programmatic assessment models that prioritize longitudinal learning, reflection and development of higher‐order cognitive skills. As part of this transition, we are exploring alternative and more meaningful forms of assessment. This qualitative study
Narin Akrawi +2 more
wiley +1 more source
BMJ Open, 2019 Objective To determine differences in the characteristics of cancer drugs designated as orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency (EMA).Design and setting Identification of all cancer drugs (initial or ...
Aaron S Kesselheim +1 more
doaj +1 more source
Is Twenty-two Months Beyond the Best Interest of the Child? ASFA\u27s Guidelines for the Termination of Parental Rights [PDF]
, 2001 This Note first discusses the legal precedents, child development theories, and policies regarding reasonable efforts and parental termination that led to the enactment of ASFA.
Hort, Katherine A.
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