A companion diagnostic is a medical device, often an in vitro device, which provides information that is essential for the safe and effective use of a corresponding drug or biological product.
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Drug repositioning: a brief overview
Published by JPP
Research and Drug DevelopmentDrug DiscoveryDrug RepurposingGenericOverview of the drug repositioning benefits both its public health benefits and its commercial value.
View this resource Bookmark this resourceThe European Medicines Agency (EMA) provides regulatory and scientific support to help acadmeics develop medicines
View this resource Bookmark this resourcea 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.
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FAIR principle for data use
Published by IRDiRC
Research and Drug DevelopmentRare DiseasePatient RegistryFAIRGuidelineLack of ultimate data use in rare disease created many silos slowing down development. The FAIR is coming to bridge this gap by proving essential guidelines for optimal data use.
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Initiatives for undiagnosed diseases
Published by IRDiRC
Research and Drug DevelopmentPatient InformationUndiagnosed Disease ProgrammeGenetic ConditionGenotype and Phenotype MatchmakingThe purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second cases
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New-born screening programs
Published by IRDiRC
Research and Drug DevelopmentRare DiseasePatient InformationGenetic ConditionNewborn Sreening ProgrammeThe purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.
View this resource Bookmark this resourceIn addition to traditional grantmaking to support people and organizations working toward our missions in Science, Education, and within our communities, the Chan Zuckerberg Initiative makes venture investments in impact-focused companies, builds tools and products that we can scale and give away for free, and supports capacity building to achieve progress across our work.
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Advisory Committee for Therapeutics (ACT) toolkit – Terms of Reference
Published by TREAT-NMD
TREAT-NMDTranslational ResearchResearch and Drug DevelopmentClinical DevelopmentPreclinical ResearchTemplatesAdvice
A decade of optimizing drug development for rare neuromuscular disorders through TACT
Published by
Advisory Committee for Therapeutics2020 marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.
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