Pediatric Neurology
VIDEO: Lobby Talks, clinical research workshops at CNS annual meeting help advance careers
In this Healio video, Ariel Maia Lyons-Warren, MD, PhD, shares resources that promoted career advancement at the Child Neurology Society annual meeting in Vancouver.
VIDEO: Conference reviews year of advances in child neurology
Ariel Maia Lyons-Warren, MD, PhD, an instructor of child neurology at Baylor College of Medicine and Texas Children’s Hospital, discusses what stood out to her at the Child Neurology Society annual meeting in Vancouver.
FDA approves Agamree for Duchenne muscular dystrophy
The FDA has approved a 40 mg/mL oral suspension of Agamree to treat Duchenne muscular dystrophy in patients aged 2 years and older, according to a release from the manufacturer.
VIDEO: 'Big emphasis' on sleep medicine, mitochondrial disorders at CNS annual meeting
Robert C. Stowe, MD, an instructor of neurology at Boston Children’s Hospital and Harvard Medical School, discusses highlights from the Child Neurology Society annual meeting in this Healio video.
Babies with spinal muscular atrophy able to sit up after 1 year of Evrysdi treatment
Genentech has announced positive results from its primary analysis of an ongoing study assessing safety and efficacy of Evrysdi in babies with pre-symptomatic spinal muscular atrophy.
Serum neurofilament light chain database effective in estimating pediatric neuro issues
Compared with absolute values, serum neurofilament light chain Z scores were linked to higher effect size metrics and more accurate estimation of persistent neuroaxonal damage in pediatric populations, per data from The Lancet Neurology.
Positive data reported from clinical trials of novel therapeutic for Dravet syndrome
Stoke Therapeutics Inc. has announced new positive data regarding its proprietary antisense oligonucleotide therapeutic to treat Dravet syndrome.
First patient dosed with electron accelerator-produced copper in neuroblastoma study
The first patient has been dosed in a clinical trial with electron accelerator-produced Cu-67, according to a Wisconsin-based company specializing in radiopharmaceuticals for therapeutic and medical imaging applications announced.
First patient dosed in phase 2 study of hypoxic ischemic encephalopathy drug
A Virginia-based biotech company has announced dosing of the first patient in a phase 2 clinical trial examining safety and efficacy of its investigational anti-inflammatory peptide, RLS-0071, to treat hypoxic ischemic encephalopathy in newborns.
Positive data reported in phase 1/2a studies of therapeutic for pediatric Dravet syndrome
A biotechnology company has announced positive safety and efficacy data from two ongoing phase 1/2a studies as well as an open-label extension study of STK-001 to treat younger individuals with Dravet syndrome.