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Alternative designs for clinical trials in rare diseases.Academic Article Why?
Efficient ways exist to obtain the optimal sample size in clinical trials in rare diseases.Academic Article Why?
Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium.Academic Article Why?
FORGE Canada Consortium: outcomes of a 2-year national rare-disease gene-discovery project.Academic Article Why?
From gene to treatment: supporting rare disease translational research through model systems.Academic Article Why?
Harmonising phenomics information for a better interoperability in the rare disease field.Academic Article Why?
Limited responsiveness related to the minimal important difference of patient-reported outcomes in rare diseases.Academic Article Why?
Rare diseases and effective treatments: are we delivering?Academic Article Why?
The Canadian Rare Diseases Models and Mechanisms (RDMM) Network: Connecting Understudied Genes to Model Organisms.Academic Article Why?
The Matchmaker Exchange: a platform for rare disease gene discovery.Academic Article Why?
A call for global action for rare diseases in Africa.Academic Article Why?
Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health Framework.Academic Article Why?
Congenital H-type tracheoesophageal fistula: A multicenter review of outcomes in a rare disease.Academic Article Why?
EpigenCentral: Portal for DNA methylation data analysis and classification in rare diseases.Academic Article Why?
The Medical Genome Initiative: moving whole-genome sequencing for rare disease diagnosis to the clinic.Academic Article Why?
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