Marco Meglio

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.

John Harsh, PhD, clinical research director, Colorado Sleep Institute, sat down at SLEEP 2023 to discuss the RESTORE study results, and why patients with narcolepsy appear to prefer once-nightly sodium oxybate.

The hold comes nearly a month after Health Canada sent PepGen a No Objection Letter to start its phase 2 CONNECT1-EDO51 trial in patients with Duchenne muscular dystrophy.

After discussions with the FDA, the study will assess 2 co-primary end points of change in Rett Syndrome Behavior Questionnaire total score and Clinical Global Impression Improvement Scale score.

Patients on twice-nightly oxybate reported more issues with inconvenience, anxiety, and feeling somewhat, quite a bit, or extremely groggy/unsteady the next morning.