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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

Increases in myelin, white matter reported at 6 months with Canavan disease gene therapy

Myrtelle Inc. has reported promising 6-month trial data for a recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease.

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March 30, 2023
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Pitolisant safe, improved symptoms of narcolepsy in children

Pitolisant safe, improved symptoms of narcolepsy in children

Treatment with pitolisant demonstrated significant efficacy in reducing excessive daytime sleepiness and cataplexy in children aged 6 years and older, according to research presented in The Lancet Neurology.

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March 28, 2023
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Gene transfer therapy safe, efficacious for children with Duchenne muscular dystrophy

Gene transfer therapy safe, efficacious for children with Duchenne muscular dystrophy

Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

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March 24, 2023
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Phase 3 interim results show casimersen safe, efficacious in Duchenne muscular dystrophy

Phase 3 interim results show casimersen safe, efficacious in Duchenne muscular dystrophy

Treatment with casimersen increased exon 45 skipping from baseline and was well-tolerated in children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

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March 22, 2023
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Givinostat improved mobility, physical function in boys with Duchenne muscular dystrophy

Givinostat improved mobility, physical function in boys with Duchenne muscular dystrophy

Treatment with givinostat was associated with improvement in mobility and physical function compared with placebo in ambulant boys with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

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February 17, 2023
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FDA approves Lamzede for non-CNS alpha-mannosidosis in adults, children

FDA approves Lamzede for non-CNS alpha-mannosidosis in adults, children

The FDA has approved Lamzede, an enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients, Chiesi Global Rare Diseases announced in a press release.

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January 30, 2023
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Impaired motor skills may indicate psychosis for kids in families with psych disorders

Impaired motor skills may indicate psychosis for kids in families with psych disorders

In children with familial high risk of schizophrenia or bipolar disorder, evidence of motor impairment skills may indicate neurodevelopmental vulnerability for psychosis.

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January 24, 2023
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FDA clears IND application for gene therapy to treat children with Rett syndrome

FDA clears IND application for  gene therapy to treat children with Rett syndrome

Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, an adeno-associated virus gene therapy candidate for treating Rett syndrome in female pediatric patients.

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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

Positive interim data announced in phase 1/2 trial of gene therapy for  Canavan disease

Myrtelle Inc. announced updated findings of the open-label phase 1/2 clinical trial of its recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease, a fatal genetic disorder in children.

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January 08, 2023
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Hassenfeld Children’s Hospital at NYU Langone names director of pediatric neuro-oncology

Hassenfeld Children’s Hospital at NYU Langone names director of pediatric neuro-oncology

Jessica Clymer, MD, has been appointed director of pediatric neuro-oncology at Hassenfeld Children’s Hospital at NYU Langone.

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