This is TREAT-NMD webpage, where it can be found relevant information how to find biomaterials for The basic scientific research being carried out on Neuromuscular Diseases. It can work also as inspiration for investigators conduting reasech in others rare diseases
View this resource Bookmark this resourceDisease 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)
the Biobanco Nacional de Enfermedades Raras (BioNER) website’s objective is to provide quality biological samples to support national and international rare diseases research. The Spanish Health Institute Carlos III (ISCIII) is the head of BioNER, being its operational and functional management and direction attributed to Instituto de Investigación de Enfermedades Raras (IIER). BioNER entered in el Registro de Biobancos with registration number B.0000886 on November 14th 2013 and the file that controls and stores the data is also registered with the Spanish Data Protection Agency (AEPD) under number 2060180217, on July 5th 2012, under the name of “Registro de Enfermedades Raras y Banco de Muestras”.
View this resource Bookmark this resourceEuroBioBank is a unique network of biobanks that stores and distributes quality DNA, cell and tissue samples for scientists conducting research on rare diseases. The network consists of 25 RD biobanks located in 11 countries.
View this resource Bookmark this resourceThis website offers a set of free biomarker commercialization tools to help researchers, small and medium sized companies (SMEs), and technology transfer offices (TTOs) tackle the highly regulated and interdisciplinary landscape of in-vitro diagnostics applicable biomarkers, i.e., markers intended to provide information on the health status of a person.
The tools guide researchers and product developers through the technology readiness levels (TRL) and remind about the technical, regulatory, and business aspects of the innovation process.
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Biomarkers in Rare Diseases
Published by International Journal of Molecular Sciences
BiomarkersRare diseasesThe focus of this article is to provide a broad platform for original research and state-of-the-art reviews on novel or established proteomic, metabolomic, or transcriptomic biomarkers that contribute to the understanding of the underlying molecular mechanisms of rare diseases and/or that can be used for the diagnosis and prognosis of disease and individuals’ responses to therapies. The collection includes eight original research papers, one commentary, and six review articles from groups investigating biomarkers for a diverse range of rare diseases.
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Predictive biomarker-based assay development in the context of drug development and lifecycle
Published by EMA
Predictive BiomarkersEMAThe proposed concept paper is intended to be developed into a guideline which will replace the reflection paper on co-development of pharmacogenomic biomarkers and assays in the context of drug development. The guideline will provide recommendations on the interface between predictive biomarker-based assays including CDx, and the development and lifecycle of a medicine.
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OECD Guidelines on Human Biobanks and Genetic Research Databases
Published by Organization for economic cooperation and development (OECD)
Human BiobankOECDThis OECD Recommendation on Human Biobanks and Genetic Research Databases (“Recommendation”) aims to provide guidance for the establishment, governance, management, operation, access, use and discontinuation of human biobanks and genetic research databases (“HBGRD”)
View this resource Bookmark this resourceIARC has developed its own Standard Operating Procedures (SOPs) for the collection, storage and shipment of biological samples. In addition, IARC provides the links of interesting biobank websites and resources.
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EATRIS Matchmaking for lab services
Published by n/a
EATRISIndustry collaborationcore facilitytechnology platformsEATRIS facilitates academic collaborations with industry. It identifIes scientific & medical expertise and find the capacity to provide access to the latest technologies and core facilities from within our technology platforms.It serves as a single point of contact, provides (legal) support and acts as a negotiator to facilitate project initiation and monitor project execution when needed. Its infrastructure consists of 115+ biomedical research institutes in 14 EU countries.
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Target Patient Value Profile
Published by IRDiRC
Research and Drug DevelopmentTarget Patient Value ProfileDrug DescriptionPatient-Centric InformationA document outlining the goals, profile and potential benefit of a specific product, addressing relevant current and future patient needs in a differentiated way. It provides accurate, up-to-date information describing the expected benefit for patients and describe the drug.
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