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Pediatric Neurology
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April 21, 2023
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Increases in myelin, white matter reported at 6 months with Canavan disease gene therapy
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March 30, 2023
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Pitolisant safe, improved symptoms of narcolepsy in children
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March 28, 2023
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Gene transfer therapy safe, efficacious for children with Duchenne muscular dystrophy
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March 24, 2023
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Phase 3 interim results show casimersen safe, efficacious in Duchenne muscular dystrophy
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March 22, 2023
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Givinostat improved mobility, physical function in boys with Duchenne muscular dystrophy
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February 17, 2023
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FDA approves Lamzede for non-CNS alpha-mannosidosis in adults, children
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January 30, 2023
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Impaired motor skills may indicate psychosis for kids in families with psych disorders
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January 24, 2023
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FDA clears IND application for gene therapy to treat children with Rett syndrome
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January 12, 2023
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Positive interim data announced in phase 1/2 trial of gene therapy for Canavan disease
News
January 08, 2023
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