Results 121 to 130 of about 3,868,024 (353)
What Is the Purpose of the Orphan Drug Act?
PLoS Medicine, 2017 Matthew Herder suggests it may be time to re-examine the purpose of the U.S. Orphan Drug Act.
M. Herder
semanticscholar +1 more source
Precision therapies for genetic epilepsies in 2025: Promises and pitfalls
Epilepsia Open, EarlyView.Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang +3 more
wiley +1 more source
Epilepsia Open, EarlyView.Abstract Objective To assess the efficacy and tolerability of fenfluramine (FFA) with concomitant potassium bromide (BR) in patients with Dravet syndrome (DS). Methods This multicenter retrospective study, conducted within the German compassionate use program, analyzed BR doses and serum levels before and after FFA initiation, adverse events (AEs ...
Milka Pringsheim +13 more
wiley +1 more source
Medicine in Drug Discovery, 2023 Only 5% of orphan diseases have approved drugs, leading to a high demand for new treatment. As a result, pharmaceutical companies have shifted their focus of drug development to orphan diseases.
Ryo Okuyama
doaj
Sick of Robots—Heterogeneous Effects of Industrial Robots on Sickness Absence
Health Economics, EarlyView.ABSTRACT This paper studies how the introduction of industrial robots affects sickness absence among workers in the manufacturing sector in Norway. We use data on the imports of industrial robots at the firm level, combined with employee‐firm linked register data, to investigate the impact of robotization on the duration of sick leave (SL).
Janis Umblijs, Kjersti Misje Østbakken
wiley +1 more source
Orphan drugs in Australia [PDF]
Expert Opinion on Orphan Drugs, 2016 The Australian orphan drugs program has the aim of encouraging research into, and the marketing of therapies aimed at the treatment of rare diseases.
openaire +2 more sources
Children Affected by AIDS: A Review of the Literature on Orphaned and Vulnerable Children [PDF]
, 2010 This paper presents a systematic review of the literature pertaining to orphans and vulnerable children in sub-Saharan Africa, with a particular focus on research in countries heavily impacted by HIV/AIDS.
Miller, Candace
core +1 more source
International Journal of Cancer, EarlyView.What's New? Multiple genetic polymorphisms characteristic of type 2 diabetes (T2D) have been associated with unfavorable prognostic outcomes in individuals with both T2D and metastatic colorectal cancer. This study evaluated the impact of T2D genetic polymorphisms on metastatic colon cancer prognosis in a cohort of non‐diabetic patients.
Raffaella Ruggiero +11 more
wiley +1 more source
The human orphan nuclear receptor PXR is activated by compounds that regulate CYP3A4 gene expression and cause drug interactions. [PDF]
, 1998 J�rgen Lehmann +5 more
openalex +1 more source
Adhesion GPCRs are widely expressed throughout the subsections of the gastrointestinal tract [PDF]
, 2012 Background: G protein-coupled receptors (GPCRs) represent one of the largest families of transmembrane receptors and the most common drug target. The Adhesion subfamily is the second largest one of GPCRs and its several members are known to mediate ...
Badiali, Luca +5 more
core +3 more sources