Results 51 to 60 of about 3,868,024 (353)
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
Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC
Hepatology, EarlyView., 2022 Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu +11 more
wiley +1 more source
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
Thirty Years of Orphan Drug Legislation and the Development of Drugs to Treat Rare Seizure Conditions: A Cross Sectional Analysis. [PDF]
PLoS ONE, 2016 Epilepsy is a serious chronic health condition with a high morbidity impairing the life of patients and afflicted families. Many epileptic conditions, especially those affecting children, are rare disorders generating an urgent medical need for more ...
Jan Henje Döring +3 more
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
Quantitative retrospective natural history modeling for orphan drug development
Journal of Inherited Metabolic Disease, 2020 The natural history of most rare diseases is incompletely understood and usually relies on studies with low level of evidence. Consistent with the goals for future research of rare disease research set by the International Rare Diseases Research ...
S. Garbade +10 more
semanticscholar +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
Innovare Journal of Life Sciences, 2020 Pharmaceutical industry is crucial for mankind and contributes in some way in socio economic development of the society through jobs, community welfare, and supply chains. Indian pharmaceutical companies are one of the largest markets in the world, estimated 10% of global production and 2% of world market.
openaire +3 more sources
Compassionate use of orphan drugs [PDF]
Orphanet Journal of Rare Diseases, 2015 EU regulation 726/2004 authorises manufacturers to provide drugs to patients on a temporary basis when marketing authorisation sought centrally for the entire EU is still pending. Individual Member States retain the right to approve and implement such 'compassionate use' programmes which companies will usually provide for free.
Jonathan C. P. Roos +3 more
openaire +3 more sources
G protein-coupled receptors not currently in the spotlight: free fatty acid receptor 2 and GPR35 [PDF]
, 2017 It is widely appreciated that G protein-coupled receptors have been the most successfully exploited class of targets for the development of small molecule medicines. Despite this, to date, less than 15% of the non-olfactory G protein-coupled receptors in
Milligan, Graeme
core +1 more source