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Rare Pediatric Disease
News
March 19, 2024
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FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy
News
February 21, 2024
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FDA grants rare pediatric disease designation to Duchenne muscular dystrophy drug
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January 17, 2024
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FDA grants rare pediatric disease designation to alpha kinase inhibitor for ROSAH syndrome
News
December 15, 2023
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First patients dosed in clinical trial of Rett syndrome gene therapy
News
October 03, 2023
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FDA removes partial clinical hold on IND for rare pediatric disease drug-device combo
News
August 09, 2023
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FDA grants rare pediatric disease designation to HG004 for inherited retinal diseases
News
July 21, 2023
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FDA grants rare pediatric disease designation to neonatal lung therapy
News
March 01, 2023
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FDA approves first oral treatment for Friedreich’s ataxia
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April 18, 2022
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Emerging therapeutics may improve outcomes in pediatric patients with rare disorders
News
January 04, 2022
1 min read
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