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.'
Neurofilament light, a marker of neuroaxonal damage, is correlated with increased levels of disease progression and clinical relapses.
Episode 39 of the AUPN Leadership Minute features Donald S. Higgins Jr, MD, of Veterans Health Administration, Albany Medical College; and Alexandra Nelson, MD, PhD, of the University of California San Francisco. [WATCH TIME: 5 minutes]
The trial will feature 60 individuals with early-stage Parkinson disease who will be randomly assigned 1:1 to either daily CVN424 or placebo for a 12-week treatment period.
The associate director of the Alzheimer’s Disease Research Unit at the Yale School of Medicine discussed the mechanism of action of ALX-001, a highly selective agent in development for neurodegenerative diseases. [WATCH TIME: 5 minutes]
In addition to myasthenia gravis, KYV-101 is being explored in other disorders like systemic sclerosis, multiple sclerosis, and lupus nephritis.
Carbidopa levodopa enteral suspension for Parkinson disease demonstrated cost-effectiveness compared to standard care, with an incremental cost-effectiveness ratio of $7711/QALY in a real-world study.
Long-term nicotinamide riboside use in patients with ataxia telangiectasia showed safety, improved motor coordination, and eye movements, suggesting potential disease attenuation.
Here's some of what is coming soon to NeurologyLive® this week.
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.
The founder of Strategic Advocacy Solutions LLC discussed the critical role of psychosocial support throughout various stages of neuromuscular diseases, emphasizing the impact of transitions on patients' emotional well-being. [WATCH TIME: 5 minutes]
Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is Parkinson disease.
Steve Hughes, MD, chief medical officer at Avidity, provided perspective on recently announced positive phase 1/2 and OLE data evaluating AOC 1001, an investigational antisense oligonucleotide, in patients with myotonic dystrophy type 1.
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.
Neurology News Network for the week ending November 11, 2023. [WATCH TIME: 3 minutes]
Patients with multiple sclerosis (MS) showed cognitive improvements with frontal theta-transcranial alternating current stimulation (tACS), suggesting its potential as a tolerable and beneficial intervention.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 10, 2023.
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.
The director of the neuromuscular diseases division at the University of Washington discussed findings from a new post hoc analysis presented at AANEM 2023 that showed that zilucoplan treatment significantly reduced fatigue in patients with myasthenia gravis. [WATCH TIME: 6 minutes]
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.
The phase 2 EMBOLD study on ATA188, an allogenic T-cell immunotherapy for non-active progressive multiple sclerosis, did not meet its primary endpoint of confirmed disability improvement after 12 months of treatment.
A retrospective study on veterans using lamotrigine found no clear association between the drug and cardiac conduction abnormalities, prompting the need for further research.
The reported neurofilament light elevation preceding confirmed disability worsening events highlighted the value of NfL as an early biomarker of disability worsening and points to the existence of different windows of dynamic central nervous system pathology.
The radiation oncologist at Cooperman Barnabas Medical Center talked about how trigeminal neuralgia can be effectively treated with stereotactic radiosurgery using the CyberKnife offering pain relief without the need for invasive surgery. [WATCH TIME: 6 minutes]
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.
The chief scientific officer at Athira Pharma talked about new preclinical results presented at the 2023 NEALS Annual Meeting on the investigational therapy ATH-1105 for patients with amyotrophic lateral sclerosis. [WATCH TIME: 5 minutes]
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.
The vice president of Innovation and New Technologies at Grifols provided perspective on the interim phase 2 findings of ABvac40, a vaccine in development for patients with Alzheimer disease. [WATCH TIME: 3 minutes]
Over a year’s worth of treatment, investigators observed consistent and persistent efficacy in pain relief, pain freedom, functional disability relief, and functional disability freedom.
The vice president of translational medicine at Cene Nanomedicine talked about a recently funded grant that is focused on inclusivity and the collection of real-world data on the efficacy of CNM-Au8 in various stages of amyotrophic lateral sclerosis. [WATCH TIME: 7 minutes]
