Results 91 to 100 of about 88,754 (289)

Preisregulierung durch Nutzenbewertung bei seltenen Erkrankungen

Monitor Versorgungsforschung
Ziel der Studie ist eine genaue Untersuchung der populationsnormalisierten Behandlungskosten von Orphan Drugs. Zu diesem Zweck wurde bereits im Jahr 2022 eine Studie zur Betrachtung der Therapiekosten von Orphan Drugs für die Jahre 2005 bis 2021 ...
Lukas Maag MSc, Dr. Ariane Höer, Dr. rer. nat. Gerrit Müller   +2 more
doaj   +1 more source

Decoding the Pathophysiology of Autoimmune Diseases—Mechanism, Triggers, and Nanotherapeutics: A Review

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, Yunhui Min, Eun‐Ju Ko, Doyoung Kwon, Rehan Khan, Young‐Ok Son   +5 more
wiley   +1 more source

Feasibility of following up gamma-hydroxybutyric acid concentrations in sodium oxybate (Xyrem®)-treated narcoleptic patients using dried blood spot sampling at home : an exploratory study [PDF]

, 2013
Background: Gamma-hydroxybutyric acid (GHB), well known as a party drug, especially in Europe, is also legally used (sodium oxybate, Xyrem (R)) to treat a rare sleep disorder, narcolepsy with cataplexy.
Hertegonne, Katrien, Ingels, Ann-Sofie, Lambert, Willy, Stove, Christophe   +3 more
core   +1 more source

Analysis of the Progression of the Representation of Female Protagonists in the Sci-Fi/Fantasy TV Shows Orphan Black and Buffy the Vampire Slayer: Girl Power, Appropriated “Masculinity” in Conjunction with Femininity, Empowered Sexuality, and the Heterosexual Script [PDF]

, 2017
Buffy Summers goes from urban Los Angeles to Sunnydale, California after she is expelled from her previous high school on the grounds of an assortment of Slayer-related incidents.
Becker, Dakota
core   +1 more source

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, Virginie Gonzalez, Lucie Beaudoin, Noémie Sénot, Corinne Miceli‐Richard, Yannick Allanore, Agnès Lehuen, Jerôme Avouac   +7 more
wiley   +1 more source

orphan drug

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.
  +4 more sources

Developing Methodology for the Creation of Clinical Practice Guidelines for Rare Diseases : A Report from RARE-Bestpractices [PDF]

, 2015
Date of Acceptance: 29/05/2015 The research leading to these results has received funding from the (FP7/2007–2013), under grant agreement n 305690 (RARE-Bestpractices project). The opinions, presented here reflect only the authors’ views.
RARE-Bestpractices Consortium
core   +1 more source

From cramming to binge‐watching: Integrating documentary‐based assessment into a pharmacology and toxicology curriculum—a qualitative study

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, Heleen van der Sijs, Floor van Rosse   +2 more
wiley   +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
core   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

British Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay, Amandine Manon, Hunter Stephens, S. Y. Amy Cheung, Bart de Keizer, Erik T. te Beek   +5 more
wiley   +1 more source