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
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.
Vutrisiran Shows Benefit Across Baseline Severities in Phase 3 HELIOS-A Study
In a recent post hoc analysis presented at the AANEM meeting, findings highlight the positive impact of vutrisiran on quality of life, disability, gait speed, and nutritional status across different neuropathy impairment score quartiles.
Treatment Inequality Issues Identified for Patients With Generalized Myasthenia Gravis
In a recent online survey of 150 participating neurologists in the US, 74.7% reported that patients with generalized myasthenia gravis found it difficult to afford their prescribed therapies, and 60.0% noted an increased likelihood of exacerbation or hospitalization for these patients.
Subcutaneous Efgartigimod PH20 Demonstrates Efficacy for Generalized Myasthenia Gravis in Open Label ADAPT-SC+ Trial
Presented at the 2023 AANEM meeting, new findings suggest that subcutaneous efgartigimod could be a valuable treatment option for patients with myasthenia gravis, with well-tolerated cycles and improved daily living activities.
Preclinical Data Shows Significant Efficacy With Low-Dose of ORX750 for Narcolepsy
Presented at the 2023 World Sleep Congress, preclinical findings highlighted ORX750's potential as a novel treatment not only for narcolepsy type 1 but also for broader sleep-wake disorders, including narcolepsy type 2 and idiopathic hypersomnia.
Takeda’s TAK-861 Shows Promise in Improving Wakefulness in Phase 1 Trial Among Healthy Men
A recent study assessing TAK-861, an orexin-type 2 receptor agonist, high and lose doses of the therapy demonstrated improvements in wakefulness compared with the placebo in healthy adult men.
NeurologyLive® Clinician of the Month Spotlight: Laura K. Stein, MD, MPH
As part of our monthly clinician spotlight, NeurologyLive® highlighted stroke expert Laura K Stein, MD, MPH, assistant professor of neurology at the Icahn School of Medicine at Mount Sinai and attending physician at the Mount Sinai and Mount Sinai Queens Stroke Centers.
FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy
With the FDA approval, vamorolone (Agamree) becomes the first-in-class dissociative steroid therapy for patients with Duchenne muscular dystrophy. Catalyst Pharmaceuticals plans to launch the treatment in the first quarter of 2024.
Remote Exercise and Physiotherapy Programs Show Benefit for Depressive Symptoms in MS
If in-person interventions are unavailable, remote forms of telerehabilitation may be considered as a solution with very little cost-effectiveness and an easy option for physiotherapists and patients, even if it is for a short period of time.
Biogen’s BIIB080 Exhibits Favorable Outcomes in Phase 1b Trial for Early Alzheimer Disease
Patients with mild Alzheimer disease who received high doses of BIIB080 demonstrated improvement in cognitive and functional outcomes compared with placebo, supporting further investigation.
AXS-05 Shows Reduction in Relapse Risk in Phase 3 ACCORD Trial for Alzheimer Disease Agitation
Findings from the trial demonstrated rapid and sustained clinical response in patients with Alzhiemer disease agitation during the open-label treatment phase and did not show any new safety signals.
Extracellular Vesicles Prove Potential Value as α-Synuclein Biomarker in Parkinson Disease
The data suggest that central nervous system-derived extracellular vesicles in the blood will likely play a key role in biomarker development, especially for patients with Parkinson disease, in the coming years.
Addressing Unmet Needs of Narcolepsy Beyond Medication
Nancy Foldvary-Schaefer, DO, FAAN, director of the Sleep Disorders Center and staff in the Epilepsy Center at Cleveland Clinic, discussed the recent advancements in narcolepsy treatments and the importance of incorporating a holistic care approach for patients.
Combining Rasagiline With Levodopa and Benserazide Hydrochloride Shows Positive Therapeutic Effects in Mid-Late Parkinson Disease
During the progression of the movement disorder, older patients with Parkinson disease may see changes in their insulin-like growth factor and serum homocysteine indices but a potential therapy could improve these factors.
FDA Allows At-Home Dosing of Intranasal Foralumab for Multiple Sclerosis
The FDA’s decision to allow at-home dosing of intransal foralumab for patients with multiple sclerosis is likely to improve patient compliance to treatment and health outcomes, according to a recent release statement.
Despite Missing Primary End Point, Pitolisant Shows Positive Outcomes in Phase 3 INTUNE Trial for Idiopathic Hypersomnia
During the open-label treatment period, 83% of patients with idiopathic hypersomnia who completed treatment saw a significant reduction in sleepiness, with an average Epworth Sleepiness Scale change of -9.4 points.
The Significance of Normal Mean Sleep Latency in Diagnosing Narcolepsy
Mark I. Boulos, MD, BSc, FRCP, CSCN, MSc, associate professor of medicine, division of neurology, Institute of Medical Science, University of Toronto, talked about a comprehensive meta-analysis aimed to determine normal mean sleep latency values to accurately help clinicians with interpreting sleep test results.
SaiLuoTong Shows Improvements in Memory and Executive Function in Phase 2 Trial of Mild Cognitive Impairment
In a recent clinical trial, patients who received SaiLuoTong showed improvements in aspects of memory and executive function including delayed episodic memory retrieval, switching between cognitive concepts, higher-level divided attention, and multitasking compared with placebo.
Optimal Dosage for Subcutaneous Ocrelizumab Uncovered in Phase 1b OCARINA I Trial
Findings from the OCARINA I study presented at the MSMilan 2023 meeting showed that a 920 mg subcutaneous dose of ocrelizumab was well-tolerated in patients with relapsing or primary progressive multiple sclerosis, with similar exposure to the FDA-approved intravenous dose.
Mesenchymal Stem Cell Therapy Shows Cognitive and Biomarker Improvements in Multiple Sclerosis
A recent analysis presented at MSMilan 2023 showed significant cognitive and biomarker improvements among patients with progressive multiple sclerosis receiving repeated intrathecal injections of autologous mesenchymal stem cells.
Satralizumab Continues to Show Long-Term Efficacy in AQP4-IgG-Seropositive NMOSD
A new analysis of the SAkuraMoon study showed that annual relapse rate remained consistently low in satralizumab-treated patients, with high proportions of patients remaining free from relapse, severe relapse, and worsening in disability.
Compliment C5 Inhibitors in NMOSD Safe Following Rituximab Use
A recent analysis of the PREVENT and CHAMPION-NMOSD found no significant differences in safety outcomes for patients with AQP4+ NMOSD who were treated with rituximab prior to starting C5 complement inhibitors in specific timeframes.
Potassium Channel Opener XEN1101 Offers Simplicity and Strong Efficacy in Seizure Control
Jacqueline French, MD, professor of neurology at NYU Grossman School of Medicine and chief medical officer of the Epilepsy Foundation, talked about findings from a newly published study assessing XEN1101 in patients with drug-resistant epilepsy.
FDA Action Update, September 2023: Approvals, Hold Lifted, and Clearance
Catch up on any of the neurology news headlines you may have missed over the course of September 2023, compiled all into one place by the NeurologyLive® team.
New Machine Learning Model Detects Mild Cognitive Impairment Through Retinal Images
A recently developed machine learning model could analyze retinal images as well as associated data of specific features to recognize patients with mild cognitive impairment.
Phase 1/2 Findings Highlight RGX-202’s Safety, Impact on Key Duchenne Biomarkers
RGX-202, a gene therapy for Duchenne muscular dystrophy, was well-tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.
PIRA Identified as the Most Common Form of Disability Accumulation in Multiple Sclerosis
Researchers proposed a harmonized definition of progression independent of relapse activity that may improve the comparability of results in current and future study cohorts, according to a newly published systematic review.
Novel Insights on Orexin Receptor TAK-994 and its Discontinued Narcolepsy Study
Elena Koundourakis, PhD, head of orexin franchise development at Takeda, talked about the phase 2 trial investigating TAK-994 in patients with narcolepsy type 1, which showed significant improvements in wakefulness, despite discontinuation because of hepatotoxicity.
Significant Care Access Disparities Found Among Older Patients With Cognitive Decline Living Alone
A recent finding from a qualitative study raises concerns about patient safety in healthcare because the system in the US is not well equipped to address the unique needs of older adults living alone with cognitive decline.
Omaveloxolone Sustains Benefit Effect in Friedreich Ataxia at 3 Years
In a cross-study analysis, omaveloxolone slowed progression in FA by at least 50% each year for 3 years compared with corresponding data from an external control group in a natural history study.
2 Clarke Drive Cranbury, NJ 08512