Isabella Ciccone, MPH

A recent study showed the efficacy of multicompartment diffusion measures in distinguishing patients with Friedreich ataxia from controls, emphasizing the potential for these metrics in tracking disease expression and progression.

Treated patients with hereditary ATTR-mediated amyloid polyneuropathy in the NEURO-TTRansform trial showed significantly lowered serum transthyretin concentrations, less neuropathy impairments, and better quality of life.

A recent 6-month cohort study presented at the 2023 AES Annual Meeting demonstrated that adjuvant cenobamate significantly reduced seizures in patients with severe refractory focal epilepsy and suggested potential benefits in hospitalization rates.

In a recent post hoc analysis of the Study C021 trial, findings support the idea that early adjustments to concomitant antiseizure medications enhance the retention of cenobamate, an FDA-approved treatment for focal seizures.

According to a recent cross-sectional study on pediatric patients with epilepsy in Western China, most patients’ caregivers had treatment access barriers, especially for making appointments, obtaining diagnosis and examination results, and having a response from the care team.

Jim Eubanks, PhD, national director of medical affairs at Octave, provided thoughts on the company’s $10 million grant from The Michael J. Fox Foundation for Parkinson’s Research and highlighted the importance of awareness for movement disorders like Parkinson disease.

Despite additional information submitted, the FDA continues to impose a clinical hold on Entrada Therapeutics' ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy.

In a new announcement, the FDA warns patients to immediately seek out medical attention if unexplained rash, fever, or swollen lymph nodes develop when using the antiseizure medications levetiracetam and clobazam.

As part of our monthly clinician spotlight, NeurologyLive® highlighted epilepsy expert Patricia C. Dugan, MD, director of the epilepsy fellowship program at NYU Langone Health.

Neurona Therapeutics’ NRTX-1001, a regenerative neural cell therapy derived from human stem cells, showed promise in potentially suppressing seizures in patients with drug-resistant mesial temporal lobe epilepsy.

In a recently published study, investigators have identified a part of the brain that may be associated with breathing failure following a seizure in patients with severe epilepsy that cannot be managed with treatment.

The FDA has scheduled an in-person meeting for December 6th to discuss the regulatory path for BrainStorm Cell Therapeutics’ NurOwn as a potential treatment for patients with amyotrophic lateral sclerosis.

A recently published study revealed that speech markers can have a high accuracy in distinguishing between neurodegenerative diseases and healthy speech, emphasizing the significance of speech analysis in disease assessment.

A recent study conducted across 11 centers globally challenged the assumption that cognitive rehabilitation and exercise synergistically benefit patients with progressive multiple sclerosis.

A recent study suggests that palliative education and telehealth improve quality of life and advance care planning among patients with Parkinson disease and related disorders.

A recently published questionnaire study showed that certain health symptoms affected epilepsy-specific health-related quality of life beyond sociodemographic and epilepsy characteristics according to responses from both caregivers and pediatric patients.

In a recent post hoc analysis, findings showed that nearly 50% of patients treated with 1 or 2 doses of midazolam experienced return to full baseline functionality in 1 hour of administration.

Group-level analyses indicated that a combination of cipaglucosidase alfa and miglustat outperformed alglucosidase alfa/placebo in various motor function and patient-reported outcome measures, with notable improvements in walking tests and quality of life aspects like 'ability to move around' and 'energy level.'

Kevin Church, PhD, chief scientific officer at Athira Pharma, discussed preclinical findings of fosgonimeton where the therapy exhibited procognitive effects among mice models in Alzheimer disease, as presented at CTAD 2023.

Brendon Yee, PhD, a respiratory and sleep physician at the Woolcock Institute of Medical Research, discussed findings from a recent phase 1 trial presented at World Sleep Congress 2023 in which ALKS 2680 demonstrated improvements in wakefulness among patients with narcolepsy type 1.

Catch up on any of the neurology news headlines you may have missed over the course of October 2023, compiled all into one place by the NeurologyLive® team.

Recent research indicated that obtaining specific variables early in established pediatric status epilepticus cases could lead to more personalized care and potentially predict refractory convulsive status epilepticus development in patients with convulsive seizures.

A recent systematic review identified that only 5% of disease-modifying therapy trials for patients with multiple sclerosis assessed fatigue as an outcome, with only 28% among 7 trials showing statistically significant results in the measurement.

Recent findings from an analysis of the CHAMPION MG study suggest that ravulizumab continues to be an effective therapy for patients with generalized myasthenia gravis, regardless of whether they had received prior IVIg treatment.

Recent research, presented at the 2023 AANEM Annual Meeting, emphasized the potential need for patient support programs targeting at-risk populations to enhance disease management and reduce acute care utilization in myasthenia gravis.

Among 67 patients with generalized myasthenia gravis studied from January 2021 to June 2023, 89.5% failed to meet clinical trial inclusion criteria, with the most common reason being a Myasthenia Gravis Activities of Daily Living score of less than 5.

In a new post hoc analysis of RAISE-XT, early responders to zilucoplan maintained their response throughout the long-term open-label extension, irrespective of baseline characteristics.

Zilucoplan demonstrated significant and sustained improvements in myasthenic fatigue when compared with the placebo in the phase 3 RAISE study and its extension trial, RAISE-XT.

The safety and tolerability of efgartigimod was consistent across different indications and doses, showing comparable rates of treatment-related adverse events with placebo in clinical trials for myasthenia gravis, primary immune thrombocytopenia, and pemphigus.

A post hoc analysis of the phase 3 RAISE study evaluating zilucoplan, a complement C5 inhibitor, showed significantly fewer patients experienced worsening and fewer requiring rescue therapy while on medication compared with placebo.