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 assistant professor at Virginia Commonwealth University provided comment on her presentation from AANEM 2023, and the need to raise awareness towards nonneuromuscular symptoms of Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
The clinical research director of the UCSF Multiple Sclerosis Center talked about challenges in accessibility for the 3 approved therapies for NMOSD caused by third-party payor resistance as well as the importance of educating clinicians about the effectiveness of inebilizumab. [WATCH TIME: 3 minutes]
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.
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]
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 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]
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.
The chief executive officer of Cognito Therapeutics detailed clinical findings from the open-label extension of the phase 2 OVERTURE study presented at the recent Clinical Trials on Alzheimer’s Disease conference. [WATCH TIME: 4 minutes]
At 40 weeks, there were no observed cases of ARIA-edema, and new microhemorrhages occurred predominantly in patients with pre-existing conditions.
The chief executive officer of Cognito Therapeutics provided an in-depth overview of the company’s gamma sensory stimulation device, its mechanism of action, and why it serves as a promising therapy for patients with Alzheimer disease. [WATCH TIME: 5 minutes]
ACU193 treatment reduced amyloid plaques in early Alzheimer disease patients, suggesting AßOs as a viable target, with safety data and ARIA analysis presented at CTAD 2023.
The use of focused ultrasound-mediated blood-brain barrier opening has the potential to advance neurotherapeutics in the treatment of Alzheimer disease and other progreesive neurological disorder impacted by that barrier.
Patients with mild Alzheimer disease who received high doses of BIIB080 demonstrated improvement in cognitive and functional outcomes compared with placebo, supporting further investigation.
The senior vice president of clinical development at Vaccinex provided commentary on a presentation from CTAD 2023 highlighting the therapeutic potential of peptinemab, an agent that’s shown success in Huntington disease, in patients with Alzheimer disease. [WATCH TIME: 3 minutes]
