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Pediatric Neurology

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October 06, 2021
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Austedo does not improve tics in pediatric patients with Tourette syndrome

Austedo did not efficaciously treat tics linked to Tourette syndrome in children and adolescents, according to results of a randomized clinical trial published in JAMA Network Open.

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October 05, 2021
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Intracranial hypertension may increase disordered eating in youth

Intracranial hypertension may increase disordered eating in youth

Young people with intracranial hypertension had an increased prevalence of disordered eating behaviors, according to study results published in Pediatric Neurology.

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September 30, 2021
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Novel gene therapy improves outcomes in children with rare neurometabolic disorder

Novel gene therapy improves outcomes in children with rare neurometabolic disorder

PTC Therapeutics Inc. released results of a 5-year analysis that showed its novel gene therapy, PTC-AADC, improved outcomes among children with aromatic L-amino acid decarboxylase deficiency.

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September 24, 2021
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Biogen to launch study of higher Spinraza dose in patients with later onset SMA

Biogen announced it intends to initiate a global phase 3b study to investigate clinical outcomes and safety of a higher dose of Spinraza in patients with later onset spinal muscular atrophy previously treated with Evrysdi.

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September 23, 2021
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Translarna delays ambulation loss in boys with Duchenne muscular dystrophy

PTC Therapeutics Inc. released results showing that treatment with Translarna delayed loss of ambulation by more than 5 years among boys with nonsense mutation Duchenne muscular dystrophy vs. standard of care alone.

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September 14, 2021
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FDA approves Briviact for young children with partial-onset seizures

The FDA has approved an expanded indication for Briviact CV tablets, oral solution and injection, for treating partial-onset seizures among children as young as 1 month.

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September 13, 2021
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CBD gel reduces seizures in children with encephalopathies

CBD gel reduces seizures in children with encephalopathies

Cannabidiol transdermal gel improved outcomes among children with developmental and epileptic encephalopathies, according to results of a nonrandomized controlled trial published in JAMA Network Open.

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September 10, 2021
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Complexity index predicts medication doses in kids with severe neurological impairment

Complexity index predicts medication doses in kids with severe neurological impairment

Children with severe neurological impairment who had higher scores on the Medication Regimen Complexity Index were more likely to receive many more doses of medications per day from their parents, according to a cross-sectional study.

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August 23, 2021
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Novel GM2 gangliosidosis treatment improves symptoms, functioning, QOL

IntraBio Inc. announced positive clinical trial results for IB1001, a pipeline drug intended to treat GM2 gangliosidosis.

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August 23, 2021
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Stroke score modified for pediatric patients correlates with severity, other outcomes

Stroke score modified for pediatric patients correlates with severity, other outcomes

The modified pediatric Alberta Stroke Program Early CT Score correlated with stroke severity, hemorrhagic transformation and 1-year outcomes among children with supratentorial ischemic stroke, according to findings published in Neurology.

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