Isabella Ciccone, Assistant Editor, NeurologyLive®, has been with the team since September 2022. Follow her on Twitter @iciccone7 or email her at firstname.lastname@example.org
Multicompartment Models of Free-Water Imaging Shows Distinctive Brain Pathology in Friedreich Ataxia
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.
Eplontersen Continues Positive Display in Phase 3 Trial for Hereditary Transthyretin Amyloidosis With Polyneuropathy
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.
Adjuvant Cenobamate Shows Significant Reductions in Seizures and Hospitalizations in Severe Refractory Epilepsy
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.
Adjusting Antiseizure Medication Dosages Improves Retention of Cenobamate
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.
Study Reveals Key Factors to Treatment Access Barriers and Medication Adherence in Pediatric Epilepsy
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.
World Movement Disorders Day: Octave’s Impact on Parkinson Disease Research
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.
FDA Declines to Lift Clinical Hold on Entrada Therapeutics’ ENTR-601-44 in Duchenne Muscular Dystrophy
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.
FDA Warns of Serious Adverse Effects for Using Levetiracetam and Clobazam in Epilepsy
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.
NeurologyLive® Clinician of the Month Spotlight: Patricia C. Dugan, MD
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.
NRTX-1001 Shows Seizure Suppression in Preclinical Drug-Resistant Focal Epilepsy Models
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.
Study Reveals Association Between Amygdala Region and Persistent Breathing Loss Following Seizures
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.
FDA Grants Meeting With BrainStorm Cell Therapeutics on ALS Therapy NurOwn
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.
Machine Learning Model Using Speech Acoustics Identifies Neurodegenerative Diseases With High Accuracy
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.
CogEx Trial Questions Efficacy of Cognitive Rehabilitation and Exercise for Progressive MS
A recent study conducted across 11 centers globally challenged the assumption that cognitive rehabilitation and exercise synergistically benefit patients with progressive multiple sclerosis.
Remote Palliative Care Intervention Yields Positive Outcomes in Parkinson Disease and Related Disorders
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.
Mental and Behavioral Health Symptoms Among Key Predictors of Epilepsy-Specific Health-Related Quality of Life
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.
Intermittent Use of Midazolam Shows Continued Favorable Profile in Seizure Clusters
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.
Switch to Cipaglucosidase Alfa/Miglustat Shows Improvement in Motor Function and Quality of Life in Late-Onset Pompe Disease
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.'
Fosgonimeton's Impact on Neuroprotection and Cognition in Alzheimer Disease
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.
Evaluating Positive Phase 1 Trial Data for Narcolepsy Agent ALKS 2680
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.
FDA Action Update, October 2023: Approvals, Hold Lifted, and Allowance
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.
Study Reveals Factors Influencing Progression of Pediatric Refractory Convulsive Status Epilepticus
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.
Impact of Fatigue Overlooked in Disease-Modifying Therapy Trials for Multiple Sclerosis
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.
Sub-Analysis of CHAMPION MG Study Reveals No Differences in Ravulizumab Treatment Based on Prior Immunoglobulin Use
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.
Study Reveals Racial Disparities in Acute Care Utilization Among Patients With Myasthenia Gravis
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.
Majority of Patients With Generalized Myasthenia Gravis Fail to Meet Clinical Trial Criteria
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.
Early Zilucoplan Treatment Maintains Long-Term Benefit in Generalized Myasthenia Gravis
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 Shows Reduction in Fatigue in the Phase 3 Trials for Generalized Myasthenia Gravis
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.
Efgartigimod Shows Consistent Safety Profile Across Immunoglobulin G-Mediated Autoimmune Diseases in Trials
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.
Zilucoplan Shows Reduction in MG Worsening in Phase 3 RAISE Study for Generalized Myasthenia Gravis
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.
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