Drug repositioning and repurposing: terminology and definitions in literature
J Langedijk, AK Mantel-Teeuwisse, DS Slijkerman… - Drug discovery today, 2015 - Elsevier
Highlights•Drug repositioning and similar terms have been a trending topic since
2004.•From a legal or regulatory perspective stimulation of drug repositioning requires …
2004.•From a legal or regulatory perspective stimulation of drug repositioning requires …
Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries
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 …
access to orphan drugs. Methods A review of the literature (1998 to 2014) was performed to …
Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare …
KL Miller, LJ Fermaglich, J Maynard - Orphanet journal of rare diseases, 2021 - Springer
Background Orphan drug designations are a useful proxy to investigate trends in rare
disease drug development. Drug developers must receive a designation before they are …
disease drug development. Drug developers must receive a designation before they are …
[HTML][HTML] Progress in rare diseases research 2010–2016: an IRDiRC perspective
HJS Dawkins, R Draghia‐Akli, P Lasko… - Clinical and …, 2018 - ncbi.nlm.nih.gov
Rare diseases by definition are conditions that affect small numbers of people. The
prevalence threshold that designates a disease as rare varies in different countries …
prevalence threshold that designates a disease as rare varies in different countries …
Glioblastoma treatments: an account of recent industrial developments
E Alphandéry - Frontiers in pharmacology, 2018 - frontiersin.org
The different drugs and medical devices, which are commercialized or under industrial
development for glioblastoma treatment, are reviewed. Their different modes of action are …
development for glioblastoma treatment, are reviewed. Their different modes of action are …
Orphan drugs: the regulatory environment
P Franco - Drug Discovery Today, 2013 - Elsevier
The definition of a rare disease is not universal and depends on the legislation and policies
adopted by each region or country. The main objective of this article is to describe and …
adopted by each region or country. The main objective of this article is to describe and …
Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives
P Song, J Gao, Y Inagaki, N Kokudo… - Intractable & rare …, 2012 - jstage.jst.go.jp
Rare diseases are an important public health issue and a challenge to medical care.
Specific legislation to encourage research of rare diseases and development of orphan …
Specific legislation to encourage research of rare diseases and development of orphan …
Innovation by patients with rare diseases and chronic needs
We provide the first empirical exploration of disease-related innovation by patients and their
caregivers. Our aims were to explore to what degree do patients develop innovative …
caregivers. Our aims were to explore to what degree do patients develop innovative …
A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can …
Background Evidence-based clinical practice is challenging in all fields, but poses special
barriers in the field of rare diseases. The present paper summarises the main barriers faced …
barriers in the field of rare diseases. The present paper summarises the main barriers faced …
MicroRNAs and long non-coding RNAs in genetic diseases
Since the discovery and classification of non-coding RNAs, their roles have gained great
attention. In this respect, microRNAs and long non-coding RNAs have been firmly …
attention. In this respect, microRNAs and long non-coding RNAs have been firmly …