Neurology

If approved, RAG-21 can offer improved outcomes in patients with amyotrophic lateral sclerosis (ALS), particularly those with aggressive subtypes.

Eladocagene exuparvovec-tneq is the first FDA-approved gene therapy for individuals with aromatic L-amino acid decarboxylase deficiency.

The study found that this patient population was about 25% more likely to have serious mental health issues after childbirth.

If approved, the investigational intravenous gene therapy will be the first disease-modifying therapy for developmental and epileptic encephalopathy (DEE).

The investigators found that the SRGAP2 and SYNGAP1 genes act together to control the speed of human synapse development, which has impacts on neurodevelopmental disease pathways.

Congenital muscular dystrophy type 1a is a severe, early-onset condition that currently has no treatment, but MDL-101 may serve as a 1-time, durable treatment option.

Therapy-related complications, in addition to financial obstacles, can complicate myasthenia gravis treatment.

The diagnostic test is a companion to vorasidenib, an isocitrate dehydrogenase inhibitor that received FDA approval this past summer.

Recognizing repeat dosing efforts is important to improving outcomes in patients with Guillain-Barré syndrome who do not respond to IVIG.

Disruptions in the salience network function are shown to influence the severity of mild behavioral impairment (MBI) symptoms.

Patients aged 7 years and older can now be treated for their cataplexy or excessive daytime sleepiness with the extended-release oral formulation.

This approval makes foscarbidopa/foslevodopa the first and only subcutaneous 24-hour infusion of levodopa-based therapy for adults with Parkinson disease.

Cardiovascular diseases have a significant impact on brain health and function.

High-resolution scans can provide a picture of a patient’s brain that typical MRIs cannot, allowing for more detailed insights into the neurological effects of COVID-19.

Results are promising, particularly because they represent the second clinical trial of a GLP-1 receptor agonist showing potential benefits for PD motor symptoms

Though both treatments come with positives and negatives, ultimately, individual patient clinical presentation and accessibility will determine the proper treatment for myasthenic crisis.

Major biomarkers for cognitive impairment were observed in hospitalized patients with neurological symptoms.

Poor sleep quality is directly related to mortality risk before adjusting for lifestyle or health-related factors, and dementia was noticeably linked to mortality risk in older White males.

Xanomeline and trospium chloride is the first in a new class, offering a new approach with selectively targeting M1 and M4 receptors.

The approval marks the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC).

Migraine is a chronic neurological disease characterized by throbbing or pulsating unilateral or bilateral headache that can last from hours to days.

The authors note this is the first study to assess cerebrovascular endothelial extracellular vesicles within a cognitive condition.

The rare disease can result in progressive neurological symptoms and organ complications.

Tocilizumab reduced relapses in 4 patients with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD).

The FDA assigned a Prescription Drug User Fee Action date is January 31, 2025.

ALZ-801 has now progressed to the phase 3 APOLLOE4 study, designed to evaluate efficacy, safety, and biomarker and imaging effects.

In a promising development in multiple sclerosis research, tolebrutinib delayed the onset of disability progression in patients with nrSPMS.

The hospital pharmacists’ role in treating patients with epilepsy starts with their place on the multidisciplinary team, which identifies appropriate drug selection in the hospital. It continues with counseling and therapy reconciliation to assure smooth transitions upon patient discharge to the community.

Despite questions and controversy around some clinical research, new understandings of the pathophysiology of the disease are pushing treatments forward.

Mirdametinib is an oral, allosteric small molecule MEK inhibitor to treat pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).