Disease or Translational Resources

In this section you will have the opportunity to explore information regarding:
Patient registries,
Natural history,
Patients’ information as organizations, toolkits for patients, new-born screening programs, undiagnosed diseases programs (UDPs), EU planned cross-border treatment
Target Patient Value Profile
Databases/Tools (ODDG)
Networks
Biobanks
Biomarkers (Development, guidelines, validation, qualification, genomic, cancer, pain therapeutics)

  1. Showing 83 results for:
  2. Research and Drug Development
  3. Disease or Translational Resources
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Advisory Committee for Therapeutics (ACT) toolkit

Published by UNEW

AdviceClinical DevelopmentPreclinical ResearchResearch and Drug DevelopmentToolkits for patientsTranslational ResearchTREAT-NMD

The Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual
registration.

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BBMRI-ERIC

Published by BBMRI-ERIC

BiobankBiobanking

BBMRI-ERIC is a European research infrastructure for biobanking. We bring together all the main players from the biobanking field – researchers, biobankers, industry, and patients – to boost biomedical research. To that end, we offer quality management services, support with ethical, legal and societal issues, and a number of online tools and software solutions. Ultimately, our goal is to make new treatments possible.

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European Medicines Agency. Qualification of Novel Methodologies for Drug Development: Guidance to Applicants (EMA/CHMP/SAWP/72894/2008). 2014.

Published by European Medicines Agency (EMA)

Drug DevelopmentEMAGuidelinePharmaceuticals 

This guidance is for qualification process addresses innovative drug development methods and tools. It will focus on the use of novel methodologies developed by consortia, networks, public/private partnerships, learned societies and pharmaceutical industry for a specific intended use in pharmaceuticals R&D. 

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Expression quantitative trait loci: present and future

Published by Philos Trans R Soc Lond B Biol Sci.

eQTLTarget ValidationTool

One of the most prominent directions has been the extensive set of studies on expression quantitative trait loci (eQTLs), namely, the discovery of genetic variants that explain variation in gene expression levels. Such studies have offered promise not just for the characterization of functional sequence variation but also for the understanding of basic processes of gene regulation and interpretation of genome-wide association studies. In this review, we discuss some of the key directions of eQTL research and its implications.

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