Pediatric Neurology News

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.

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.

Visual and auditory evoked potentials showed promise as an objective measurement of Rett syndrome severity, according to a study published in Annals of Neurology.

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.

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.

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.

Individuals with familial cerebral cavernous malformation had a high incidence rate of seizures, according to study findings published in Neurology.

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

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.

The FDA placed a clinical hold on a phase 3 study designed to evaluate elivaldogene autotemcel, an investigational gene therapy, for the treatment of younger patients with cerebral adrenoleukodystrophy.