Article

The LORIS MyeliNeuroGene Rare Disease Database for Natural History Studies and Clinical Trial Readiness

2021-05-06

Abstract excerpt

<title>Abstract</title> <p><bold>Background:</bold> Rare diseases are estimated to affect 150-350 million people worldwide. With advances in next generation sequencing, the number of known disease-causing genes has increased significantly, opening the door for therapy development. Rare disease research has therefore pivoted from gene discovery to the exploration of potential therapies. With impending clinical tri...

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Literature Corpus work: 6cd01c9a-5202-58b3-a256-08fb60ac3a72
DOI: 10.21203/rs.3.rs-491571/v1

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