Isabella Ciccone, MPH

A recently initiated phase 1/2 trial of NS-050/NCNP-03 will assess patients with Duchenne muscular dystrophy on dystrophin production, muscle strength, mobility, and functional exercise capacity.

Catch up on any of the neurology news headlines you may have missed over the course of November 2024, compiled all into one place by the NeurologyLive® team.

The assistant professor of neurology at the Icahn School of Medicine at Mount Sinai provided an overview of a session on exercise and migraine presented at the 2024 Scottsdale Headache Symposium. [WATCH TIME: 5 minutes]

Findings from a phase 2/3 study demonstrated the efficacy and safety of a higher dose regimen of nusinersen in treating spinal muscular atrophy in both treatment-naïve and previously treated patients.

Shalini Paruthi, MD, FAASM, a spokesperson for the American Academy of Sleep Medicine, provided a clinical overview on restless leg syndrome for diagnosing and tailoring treatment for patients with the condition.

As part of our monthly clinician spotlight, NeurologyLive® highlighted epilepsy expert Stephanie C. Randle, MD, MS, director of the Tuberous Sclerosis Complex Clinic at Seattle Children's Hospital.

A recent multicenter study showed that dried blood spot testing for neuromyelitis optica spectrum disorder provided high accuracy and practicality in resource-limited settings.

A new study revealed stark racial and socioeconomic disparities in pediatric neuromyelitis optica spectrum disorder outcomes, underscoring the need for targeted interventions.

The pediatric neurologist at Duke Health and chief executive officer at Theranica talked about a remote electrical neuromodulation wearable that showed promising results for the treatment and prevention of migraine in pediatric patients. [WATCH TIME: 5 minutes]

The vice president of scientific engagement at the Alzheimer's Association discussed advancements in blood biomarker tests, their use in specialty care, and the development of clinical guidelines for broader integration.

Jessica Langbaum, PhD, the senior director of research strategy at Banner Alzheimer’s Institute, discussed the challenges and opportunities in improving diversity in Alzheimer disease clinical trials.

Findings from a recently published study suggest patients with Parkinson disease can reduce postural sway as efficiently as neurologically healthy patients when performing horizontal saccades.

The professor of neurology and radiology at University of California, San Francisco, discussed the potential of blood-based biomarkers in Alzheimer treatment eligibility, highlighting current limitations and the importance of safety monitoring. [WATCH TIME: 7 minutes]

The neurologist in the Headache Center at Cleveland Clinic shared her post perspective on the 2024 AHS Scottsdale meeting which highlighted advancements in headache medicine and the growing impact of the field on patient care. [WATCH TIME: 5 minutes]

The phase 2 DIMENSION study results showed that SAGE-71 was well-tolerated but did not meet its primary end point, leading to Sage Therapeutics’ decision to discontinue its development.

The dean and chief academic officer at the University of Miami Miller School of Medicine discussed the university’s investment in computational biology to advance aging and neuroscience research. [WATCH TIME: 5 minutes]

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine talked about the ongoing evolution of multiple sclerosis diagnostic criteria and the importance of comprehensive testing to guide early intervention and improve outcomes. [WATCH TIME: 5 minutes]

According to the company, VQ-101 is the first small molecule to show over 75% activation of the lysosomal enzyme GCase in humans, with phase 1 results in healthy volunteers indicating tolerability and exposure levels that support once-daily dosing.

Initial findings showed significant improvements from baseline in the disease-specific, patient-reported quality of life scores in both the high and low dose AAV-GAD groups, with no significant change in the sham group at 26 weeks.

Nerivio, a nondrug option for acute migraine attacks and prevention of migraine with or without aura, has been FDA-approved for several years in adolescents aged 12 and older as well as adults.

A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine discussed the newly updated multiple sclerosis criteria, highlighting their focus on probabilistic assessment, advanced technology integration, and early intervention to improve patient care.

The professor of neurology and radiology at University of California, San Francisco talked about how the new appropriate use recommendations of donanemab aims to safely integrate this anti-amyloid antibody into the clinical practice. [WATCH TIME: 7 minutes]

Despite advances in understanding and treating Long COVID, many questions about its mechanisms, susceptibility, and varied recovery patterns remain unresolved, underscoring the need for continued research.

The CEO at C2N Diagnostics talked about the clinical utility of the company’s PrecivityAD2 test, which uses the p-tau 217 biomarker and amyloid-β measurements, to increase diagnostic confidence and influence treatment decisions in Alzheimer disease. [WATCH TIME: 7 minutes]

The chief operating officer at Quanterix Corporation talked about pioneering ultra-sensitive blood biomarkers for Alzheimer clinical diagnosis, aiming to enhance detection of brain-derived tau and amyloid status with innovative assays. [WATCH TIME: 4 minutes]

Catch up on any of the neurology news headlines you may have missed over the course of October 2024, compiled all into one place by the NeurologyLive® team.

Nina Miolane, PhD, and Amy Kuceyeski, PhD, the codirectors of the Ann S. Bowers Women's Brain Health Initiative AI Core, provided their commentary on the challenges and progress made in women’s neurology.

In previously reported data, AOC 1020 demonstrated a consistent reduction in double homeobox 4 regulated genes among patients with facioscapulohumeral muscular dystrophy at 4 months.

The head of global clinical development for immunology/bone at UCB talked about findings presented at CTAD 2024 from the phase 2 trial assessing bepranemab in Alzheimer disease. [WATCH TIME: 3 minutes]