Marco Meglio, Assistant Managing Editor for NeurologyLive, has been with the team since October 2019. Follow him on Twitter @marcomeglio1 or email him at [email protected]
Marisa Whalen, PharmD, associate director of Medical Affairs at Jazz Pharmaceuticals, discussed presentations at SLEEP 2023 on the real-world burdens of idiopathic hypersomnia and the smooth transition to low sodium oxybate in patients with narcolepsy.
In a phase 3 study, rozanolixizumab demonstrated statistically significant differences vs placebo on primary and secondary outcomes over a 43-day treatment period.
At 6 months, a larger proportion of patients on standard of care experienced unfavorable outcomes, as demonstrated by modified Rankin Scale scores greater than 3, in comparison with lumbar drainage.
In addition to increased relapse rates, use of fingolimod was associated with a 49% higher risk for disability accumulation in comparison with ocrelizumab.
Following the positive results, uniQure expects to initiate a third cohort in the ongoing trial to further investigate both low and high doses of AMT-130 in combination with perioperative immunosuppression.
In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.
In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.
High and low doses of frexalimab resulted in reductions of new or enlarging T2 lesions and total T1 gadolinium-enhancing lesions over a 3-month period.
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Natalie Goedeker, CPNP. [LISTEN TIME: 12 minutes]
Compared with placebo, inebilizumab-treated participants showed attenuated biomarker elevation during attacks and reduced biomarker levels over time in the absence of adjudicated attacks.
Marketed as Elevidys, the gene therapy is approved for ambulatory pediatric patients aged 4 to 5 with Duchenne based on expression of micro-dystrophin.
CHALLENGE-MIG, a 3-month study, is the first trial to compare the efficacy of 2 previously approved calcitonin gene-related peptide antagonists in the treatment of migraine.
Biomarker data at 49 weeks continued to demonstrate that DNL310 enabled rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers.
Over a 12-week period, PTC518 showed a safe and tolerable profile, with significant reductions in mutant huntingtin protein and an overall trend towards lowering of neurofilament light levels.
At the 2023 AHS Annual Meeting, the chief of headache at UT Austin Dell Children’s Hospital discussed her presentation on a new educational intervention to improve knowledge of pediatric headache.
The basis for the FDA’s decision came from the phase 3 ADAPT-SC study, in which subcutaneous efgartigimod showed a slightly better ability to reduce immunoglobulin compared with its previously approved intravenous formulation.
In one of the first phase 3 trials of primary mitochondrial myopathy, elamipretide failed to meet its primary end points; however, a subgroup of patients with nuclear DNA defects saw improvement in 6-minute walk test, raising further questions.
Walter Koroshetz, MD, director of the National Institutes of Neurological Disorders and Stroke at the NIH, provided perspective on the advances in migraine treatment, and the next steps in research needed over the coming years.
Data showed similar rates of discontinuation because of adverse events between those with and without CGRP monoclonal antibodies, as well as no serious AEs observed for those on concomitant medication.
Compared with the overall population, outcomes for the coprimary end points of pain freedom and freedom from most bothersome symptom at 2 hours postdose remained superior in rimegepant-treated individuals.
Atogepant 60 mg once daily group had significantly larger reductions in mean monthly migraine days across weeks 1-12 and weeks 9-12, with no statistical difference in treatment-emergent adverse events or all-cause discontinuation.
Demographic factors of male sex, non-White race, and having a primary language other than English were associated with a lower odds of being diagnosed with migraine.
Patients on candesartan saw statistically significant reductions in the mean frequency of monthly headache days, including moderate to severe headache days.
When comparing galcanezumab, fremanezumab, and erenumab, 3 FDA-approved CGRP medications for preventive migraine, time to switch was lowest for erenumab and similar for galcanezumab and fremanezumab.
A reduction in microglial activation was observed on the PET scans of most patients included. A phase 2 trial of the therapy was announced earlier this year and is still on track to begin sometime in the fall of 2023.
Originally approved under the accelerated approval pathway, the promising efficacy observed in the study will prompt a future regulatory submission for traditional.
Over a 39-day period, treatment with SCI-110 resulted an average reduction of 23% in agitation among patients with Alzheimer disease.
The professor of neurology at UMass Chan School of Medicine provided perspective on the benefits of pushing back school start times based on what research has shown.
Robert A. Hauser, MD, MBA, director of the Parkinson’s and Movement Disorders Center at the University of South Florida, provided insight on the therapeutic potential of IPX203, and why it adds flexibility to the treatment of Parkinson disease.
In a secondary analysis of a double-blind trial, mazindol extended-release outperformed placebo on clinician and patient scales of cataplexy severity and excessive daytime sleepiness.
