Marco Meglio

Capsida’s CAP-002 gene therapy, currently advancing through IND-enabling studies, aims to address the unmet needs of patients with STXBP1-related developmental and epileptic encephalopathy by stably supplementing STXBP1 protein.

The phase 1 study, which is currently recruiting, includes a 4-week screening period, a 52-week treatment period, and a 4-year long-term extension to continue to test the effects of HG202.

Italo Linfante, MD, director of interventional neuroradiology at Baptist Health Miami Neuroscience Institute, discussed the importance of brain aneurysms in the context of neurology.

Judith Thompson, PharmD, MPH, CPHQ, rare disease population health strategy lead at UCB, discussed the association between social determinants of health and delayed diagnosis/misdiagnosis in myasthenia gravis.

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 on PIRA (progression independent of relapse activity) in multiple sclerosis.

The double-blind, placebo-controlled trial will include 200 patients with Angelman syndrome who will be followed over a 12-month period, with an additional open-label extension after.

David Chernoff, MD, chief medical officer at SetPoint Medical, provided clinical insight on a new pilot study intended to assess the therapeutic potential of a rechargeable neurostimulation aimed to reducing demyelination in relapsing multiple sclerosis.

Despite not meeting its primary end point, treatment with GV1001 showed more promising results in patients with progressive supranuclear palsy-Richardson’s syndrome over a 24-week treatment period.

M. Scott Perry, MD, an expert in epileptic disorders and board president of PERC, provided insights on the group's mission and goals, its structure and membership, and its 60+ ongoing research projects.

The cAPPricorn-1 Phase 2 trial will assess mivelsiran’s efficacy in CAA, focusing on reducing the annualized rate of new cerebral microbleeds over 24 months.

Diego Torres-Russotto, MD, chair of neurology at Baptist Health Miami Neuroscience Institute, discussed the Miami Neuroscience Symposium’s focus on addressing the community’s neurological needs, balancing content delivery and fostering multidisciplinary collaboration.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

The director of the myasthenia gravis clinic at Yale University discussed the therapeutic potential of inebilizumab, an FDA-approved treatment for NMOSD, in myasthenia gravis, based on data from the phase 3 MINT study.

The NDA includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.

Using a Brain-Chip model, investigators demonstrated that pepinemab can inhibit or reverse damage from toxic alpha-synuclein aggregates, with similar results for amyloid-ß expected soon.

Over a 48-week treatment period, once daily blarcamesine slowed clinical decline in patients with early-stage Alzheimer disease, with even more pronounced effects in pre-specified common SIGMAR1 wild type group.

At week 25 of treatment with trontinemab, patients demonstrated decreases in CSF total tau, CSF p-tau181, and CSF neurogranin.

Bruce Leuchter, MD, president and chief executive officer at Neurvati Neurosciences, provided clinical insight on the newly initiated Astroscape trial of radiprodil for the treatment of tuberous sclerosis complex and focal cortical dysplasia type II.

Amy Tsou, MD, MSc, program director at the National Institutes of Neurological Disorders and Stroke, discussed the increasing cost of prescription drugs in the US and its impact on healthcare providers, including those who treat patients with neurologic conditions.

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 on GBA1-associated Parkinson disease.

Over a 24-week period, comprising both the core study and extension trial, the incidence of treatment-emergent adverse events was infrequent, with less occurring over the long-term extension.

Ahead of International LGS Awareness Day, Melanie Huntley, PhD, a data scientist for Roche and mother of child with Lennox-Gastaut syndrome, shed light on the realities of living with LGS, emphasizing the need for greater awareness of the condition’s impact.

A subgroup analysis of the phase 3 Clarity AD open-label extension study showed a 14% increased amyloid plaque removal with subcutaneous lecanemab vs intravenous administration, the administration for which it was FDA-approved for.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lawrence Robinson, MD. [LISTEN TIME: 15 minutes]

LX1001, an AAV gene therapy, was considered safe and well tolerated, with no events of amyloid-related imaging abnormalities (ARIA) observed.

In total, more than half of the screened population failed by not meeting plasma p-tau217 criteria and of the remaining, most participants were not in the intermediate tau PET range.

Insulin treatment significantly increased beneficial plasma biomarkers, including SNAP25, SMOC1, BDNF, and VCAM1.

The rare disease population health strategy lead at UCB provided clinical insight on some of the unique challenges of diagnosing myasthenia gravis, and some of the early signs clinicians and non-specialists should look out for. [WATCH TIME: 3 minutes]

The multicenter, randomized, double-blind, placebo-controlled, parallel-group, event-driven trial will use time to clinical progression, defined as an increase in Clinical Dementia Rating score, as the primary end point.