a list of 1415 rare diseases that are related to EMMA strains. Please use the search bar to search for rare diseases, gene name, ORPHAcode or EMMA strain.
View this resource Bookmark this resourcePreclinical Studies
In this section you may find relevant information regarding the safety, efficacy and toxicology of the drugs actually used for Rare Diseases as well as:
Good Laboratory Practices (GLPs),
Animal models: juvenile, humanised animal models, disease specific animal models and documents that may support you to correctly use animal models from ethical points of view.
Proof of principle
In silico modelling
The Vienna Drosophila Resource Center (VDRC) is part of the Vienna Biocenter Core Facilities (VBCF), a publicly funded, non profit research infrastructure.
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EATRIS Regulatory support
Published by n/a
Scientic adviceEATRISRegulatory SupportOrphan Drug DesignationThrough a combination of in-house and external partnerships with a range of regulatory experts and groups, EATRIS can provide regulatory support for most types of product including: Expert opinion, Orphan Drug Designation and Scientific Advice application at the EMA, Pre-clinical and clinical plan development, Informal scientific advice with selected national competent authorities, for highly complex projects,
access to EATRIS Regulatory Database (free of charge) that contains information about the regulatory requirements, guidelines and legislations from 27 EU countries (as well as Norway, Switzerland, Turkey and Israel) regarding drug and medical device development derived from the application of European legislation.
ICH: Safety
Published by International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)EMA
Photosafety EvaluationTherapeutic Area-SpecificImmunotoxicology StudyToxicokinetics and PharmacokineticsSafety Pharmacology StudyBiotechnological ProductReproductive ToxicologyRepeat-Dose ToxicityNonclinical Safety in Paediatric MedicineCarcinogenicity StudyGenotoxicity StudyThe European Medicines Agency publishes scientific guidelines on human medicines that are harmonised by the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH)
View this resource Bookmark this resourceThe European Medicines Agency publishes scientific guidelines on human medicines that are harmonised by the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH).
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FDA Qualification process – Drug Development Tools
Published by IRDiRC
Clinical DevelopmentResearch and Drug DevelopmentRegulatory AffairsEndpointBiomarkerAnimal ModelQualification ProcessDrug Development ToolCenter for Drug Evaluation and ResearchThis guidance provides a framework for interactions between the Center for Drug Evaluation and Research (CDER) and the entity proposing the DDT for qualification (the submitter). It also explains the kinds of data that should be submitted to support qualification of a DDT and creates a mechanism for CDER’s formal review of the data to ultimately qualify the DDT.
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