Results 21 to 30 of about 1,419,402 (298)
QJM, 2006 Hughes et al .1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive.
McCabe, C. +3 more
openaire +3 more sources
A novel panel of mouse models to evaluate the role of human pregnane X receptor and constitutive androstane receptor in drug response [PDF]
, 2008 The pregnane X receptor (PXR) and the constitutive androstane receptor (CAR) are closely related orphan nuclear hormone receptors that play a critical role as xenobiotic sensors in mammals.
Zevnik, Branko +13 more
core +1 more source
Analysis of patient access to orphan drugs in Turkey
Orphanet Journal of Rare Diseases, 2021 Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases.
Güvenç Koçkaya +7 more
doaj +1 more source
Medicinski pregled, 2013 Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon +4 more
openaire +3 more sources
Pilot and Feasibility Studies, 2018 Objective To examine the feasibility, acceptability, and preliminary effects of Hatha yoga on oxidative stress, motor function, and non-motor symptoms among individuals with Parkinson’s disease (PD).
Corjena Cheung +8 more
doaj +1 more source
Precision Medicines’ Impact on Orphan Drug Designation
Clinical and Translational Science, 2019 The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration.
Christine M. Mueller +2 more
doaj +1 more source
Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia [PDF]
Hospital Pharmacology, 2016 Background/Objective: Orphan drugs are medicinal products which treat life-threatening or chronically debilitating diseases which affect small patient populations.
Pejčić Ana V., Iskrov Georgi G.
doaj +1 more source
Investigating the landscape of US orphan product approvals
Orphanet Journal of Rare Diseases, 2018 Background The Orphan Drug Act was enacted in 1983 to encourage the development of drugs for rare diseases. Previous research has attempted to examine the impact of the Act by assessing either the number of orphan designations that have been granted or ...
Kathleen L. Miller, Michael Lanthier
doaj +1 more source
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. [PDF]
PLoS ONE, 2015 OBJECTIVE:To review existing regulations and policies utilised by countries to enable patient access to orphan drugs. METHODS:A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we
Todd Gammie +2 more
doaj +1 more source
Angewandte Chemie, EarlyView.State‐of‐the‐art synthesis of radiolabelled oligonucleotides involves multi‐step synthesis with high costs, extended timelines and significant radioactive waste generation. Herein, a dual‐methyltransferase platform enables late‐stage, site‐specific labelling, overcoming these limitations through reduced costs, faster turnaround and minimal radioactive ...
Christopher R. B. Swanson +3 more
wiley +2 more sources