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The LORIS MyeliNeuroGene rare disease database for natural history studies and clinical trial readiness
BACKGROUND: 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 disc...
Autores principales: | , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8299589/ https://www.ncbi.nlm.nih.gov/pubmed/34301277 http://dx.doi.org/10.1186/s13023-021-01953-8 |