New therapeutic modalities for rare diseases
Genetic Diseases affect about 2 % to 3 % of all live births. It has been reported that genetic disorders can account for up to 22 % of infant deaths. Despite this significant effort to create new therapies, treatment is available only for about 5 % of these diseases due to undiscovered etiology or r...
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| Format: | Article |
| Language: | English |
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Elsevier
2024-03-01
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| Series: | Global Pediatrics |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2667009724000022 |