Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jval.2014.08.1738DOI Listing

Publication Analysis

Top Keywords

top orphan
4
orphan drugs
4
drugs availability
4
availability pricing
4
pricing reimbursement
4
reimbursement slovakia
4
slovakia 2005-2012
4
2005-2012 review
4
top
1
drugs
1

Similar Publications

The role of epigenetics and chromatin in the maintenance of postmitotic neuronal cell identities is not well understood. Here, we show that the histone methyltransferase Trithorax (Trx) is required in postmitotic memory neurons of the Drosophila mushroom body (MB) to enable their capacity for long-term memory (LTM), but not short-term memory (STM). Using MB-specific RNA-, ChIP-, and ATAC-sequencing, we find that Trx maintains homeostatic expression of several non-canonical MB-enriched transcripts, including the orphan nuclear receptor Hr51, and the metabolic enzyme lactate dehydrogenase (Ldh).

View Article and Find Full Text PDF

Background: Forecasting future public pharmaceutical expenditure is a challenge for healthcare payers, particularly owing to the unpredictability of new market introductions and their economic impact. No best-practice forecasting methods have been established so far. The literature distinguishes between the top-down approach, based on historical trends, and the bottom-up approach, using a combination of historical and horizon scanning data.

View Article and Find Full Text PDF

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.

View Article and Find Full Text PDF

Background: Pain is the leading cause of disability and reduced quality of life worldwide. Despite the increasing burden for patients and healthcare systems, pain research remains underfunded and under focused. Having stakeholders identify and prioritize areas that need urgent attention in the field will help focus funding topics, reduce 'research waste', improve the effectiveness of pain research and therapy and promote the uptake of research evidence.

View Article and Find Full Text PDF

GPR55, an orphan GPCR, holds significance in the context of neurological disorders and its activation is important for regulating neurological responses. This has led us to uncover and explore a set of ligands which may act as potential agonists. We have predicted the GPR55 structural model followed by molecular docking, dynamics simulation studies, free energy landscape and MMPBSA analyses to study the dynamic nature of GPR55 in the presence of different ligands.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!