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.
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 3, 2023.
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.
A study on patients with Friedreich ataxia showed that cervical spinal cord cross-sectional area is a potential biomarker for disease progression, particularly at C1 level.
The professor of neurology at Cedars-Sinai Medical Center talked about findings from the ADHERE trial assessing efgartigimod, a human IgG1 antibody Fc fragment, as a treatment for patients with chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 6 minutes]
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Alfonso Fasano, MD, PhD. [WATCH TIME: 25 minutes]
A retrospective study presented at the 2023 AANEM meeting revealed that social determinants of health are linked to poorer treatment outcomes in myasthenia gravis patients, emphasizing the need for identifying and supporting at-risk individuals.
In the phase 3 TOLEDO trial, treatment with SPN-830 resulted in significantly greater OFF time for patients with Parkinson disease in comparison with those on placebo.
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.
Elevated creatine kinase levels were not found to be a prognostic factor for the development of PASC in encephalopathic COVID-19 patients, according to a retrospective chart review analysis.
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.
The division director of Neuromuscular Medicine & EMG at USF Health provided an overview of the open-label findings from ADAPT-SC, a phase 3 study assessing subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 3 minutes]
Vutrisiran and patisiran treatment led to noticeable reductions in neurofilament light, indicating a potential biomarker of treatment response in hATTR amyloidosis patients.
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.
The phase 3 PROPEL study demonstrated the long-term effectiveness of cipaglucosidase alfa/miglustat in late-onset Pompe disease, with improved 6-minute walk distance and lung function.
The senior director and global compound development team leader at Janssen discussed research on nipocalimab, an investigational agent for patients with myasthenia gravis that's shown promising data to this point. [WATCH TIME: 2 minutes]
latozinemab treatment for C9orf72-associated frontotemporal dementia showed no significant impact on disease progression, although the treatment was generally safe and well-tolerated.
The senior director and global compound development team leader at Janssen talked about the importance of biomarkers in detecting an earlier diagnosis of myasthenia gravis and the significant unmet medical need for better treatment options. [WATCH TIME: 3 minutes]
David Greeley, MD, chief medical officer at AriBio and study author, provided commentary on the mechanism of action of AR1001, its promising effect on ptau181, and the next steps in its development.
The vice-chair for research at Cleveland Clinic’s Neurological Institute discussed the way therapies are currently assessed for progressive MS and the need for disease-specific biomarkers. [WATCH TIME: 3 minutes]
Recent research has suggested a role for thyroid hormones and their receptor, thyrotropin, in muscle regeneration and development, making a case for further investigation in DMD.
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.
Over a 52-week treatment period, SRP-9001 demonstrated robust evidence for a clinically meaningful benefit across several prespecified functional secondary end points in comparison with placebo.
Jeffrey Cohen, MD; Sarah Anderson, PharmD, NBC-HWC; and Alicyn Magruder, PharmD, BCACP, MSCS, share their final thoughts on the future landscape of clinical MS care in the biosimiliars era. [WATCH TIME: 12 minutes]
In a sample of more than 750 patients with migraine, clinician specialty had no impact on preventive prescription, with small differences observed in the choice of tricyclic antidepressants.
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.
Results showed that the relative reduction (RR) in CDR-SB at end-of-study was moderately correlated with difference between treated and placebo in Centiloid change.
START will assess the potential of delaying non–vitamin K oral anticoagulant therapy following stroke related to atrial fibrillation in a large cohort of patients
Here's some of what is coming soon to NeurologyLive® this week.
The group continues discussion about patient communication needs, and Sarah Anderson, PharmD, shares resources that the National MS Society offers to both patients and providers. [WATCH TIME: 9 minutes]
