Neurology Alert – June 1, 2024
June 1, 2024
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Novel Gene Therapy for Giant Axonal Neuropathy
The authors conducted a Phase I trial of a gene editing approach delivered by adeno-associated virus vector carrying a codon-optimized human giant axonal neuropathy transgene. The transgene targets anterior horn motor neuron bodies and sensory neurons in the dorsal root ganglia with the goal of repairing peripheral nerves and their axons. Each patient received a single intrathecal injection of the investigational agent. At one year, patients had less progression of disease compared to pretreatment intervals.
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Seizures in Adolescents: Motor or Nonmotor?
The rate at which motor vs. nonmotor seizures are recognized in adolescents is unknown. This retrospective analysis demonstrates a high rate of misrecognition of nonmotor seizures among adolescents presenting to an emergency department and frequent misrecognition of any nonmotor seizures in those presenting with a first-time motor seizure.
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Risk of Autism After Prenatal Topiramate, Valproate, or Lamotrigine Exposure
The incidence of autism spectrum disorder was higher among children with prenatal exposure to the topiramate, valproate, and lamotrigine than the risk in the general population. However, after adjustment for indication of the medication (epilepsy) and other confounders, an increased risk was only observed in children with prenatal exposure to valproate.
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Brentuximab Neuropathy
Brentuximab, an antibody-drug conjugate for the treatment of lymphomas, carries a high risk of inflammatory sensory and motor neuropathies.
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Results from GLP-1 Receptor Agonists in Early Parkinson’s Disease
This paper demonstrated that after one year of treatment, lixisenatide resulted in less progression of motor disability. Longer studies are needed to determine if this represents true disease modification.