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Rare Pediatric Disease
News
November 13, 2020
3 min read
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Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy
News
September 26, 2020
1 min read
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Innova Therapeutics receives rare pediatric disease designation for osteosarcoma treatment
News
August 26, 2020
1 min read
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FDA grants rare pediatric disease designation to cell therapy for sickle cell disease
News
January 31, 2020
1 min read
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FDA grants rare pediatric disease designation for QR-421a in retinitis pigmentosa
News
January 10, 2020
1 min read
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FDA grants rare pediatric disease designation to gene therapy for sickle cell disease
News
October 23, 2019
1 min read
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ProQR announces rare pediatric disease designation, phase 1/2 results for sepofarsen
News
October 21, 2019
1 min read
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