Isabella Ciccone, MPH

As a recap from AAN 2023, get caught up on some of the latest news in neurology as the NeurologyLive® team shares some of our data updates.

In April 2023, NeurologyLive® is spotlighting a clinician in neuromuscular diseases, Michelle Mauermann, MD, chair of the division of neuromuscular medicine at Mayo Clinic.

Inhibition of serum free complement 5 with ravulizumab was sustained throughout the treatment period according to a pharmacokinetics and pharmacodynamics analysis.

Minoryx's leriglitazone showed a decrease in matrix metalloproteinase-9 concentrations in pediatric patients with cerebral adrenoleukodystrophy, according to a 24-week analysis.

The treatment was superior in preventing on-trial relapse in both the monotherapy and immunosuppressive therapy groups compared with placebo in the CHAMPION-NMOSD trial.

Higher rates of no evidence of disease activity and no 24-week confirmed disability progression or relapses were observed in patients that continued with ocrelizumab.

Findings showed a significant reduction in mean monthly migraine days with atogepant 60 mg once daily for patients with episodic migraine compared with placebo across a 12-week period.

Zavegepant, a calcitonin gene-related peptide (CGRP) receptor antagonist, provided onset of pain relief as early as 15 minutes postdose and sustained benefits to 48 hours post dose.

An interim analysis showed no reduction in T-cell counts and their subsets as a consequence of B-cell depletion in the first year of treatment with ocrelizumab.

Updated results to the phase 3 CHAMPION-NMOSD trial of ravulizumab showed significantly lower HAI score worsening following treatment.

Resilience was positively correlated with quality of life in patients with narcolepsy type 1, according to a recent published study.

Andrew Siderowf, MD, director of the Parkinson disease and movement center at the University of Pennsylvania, talked about available therapeutics and potential future therapies in research for Parkinson disease.

Sheryl Lapidus, MA, senior director of patient advocacy at Horizon Therapeutics, talked about the findings from a recently conducted patient survey on NMOSD and the importance of advocacy in the field.

CSL Behring's subcutaneous immune globulin is the first and only immune globulin available in prefilled syringes for patients with primary immunodeficiency or chronic inflammatory demyelinating polyneuropathy.

Avantis is a platform device by Reach Neuro that empowers poststoke patients to regain use of their arm and hand, strengthening their muscle activity without imposing unintended movements.

The oral treatment, marketed as Rizafilm, is a disintegrating film formulation of the 5-HT1 receptor agonist and is bioequivalent to Merck’s Maxalt-MLT.

Findings from a recent published study observed lower quality of life in Black, Hispanic, and Asian patients with Parkinson disease compared with White patients.

New research confirms a key disease pathology identified through examining spinal fluid in patients with Parkinson disease.

Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.

Positive phase 2 data on CVN424, NETSseq platforms, and CVN417, a novel treatment for Parkinson disease, were presented at AD/PD 2023 International Conference.

New data demonstrated the potential of a new class of oral, small molecule inhibitors that target the fleeting intermediates at the core of neurodegeneration in Parkinson disease.

VO659, the only clinical candidate targeting the CAG repeat expansion that causes all polyglutamine diseases, is designed to reduce mutant HTT and spare wildtype HTT.

Michael Levy, MD, PhD, associate professor at Harvard Medical School, spoke about the breakthrough of drugs for NMOSD and the challenges patients face to receive treatment.

Evidence from a nationwide healthcare system data in South Korea showed that narcolepsy is closely related to systemic autoimmune diseases, particularly those related to HLA genes.

Robin Ryther, MD, PhD, director of the Rett Spectrum Clinic at Washington University in St. Louis School of Medicine, shared her response to the recently approved therapy trofinetide for Rett syndrome.

By the end of the open label long-term extension study, BIIB080 reduced aggregated tau pathology in all dose groups assessed across all brain composites.

A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.

Investigators exercised caution when considering administration of antithrombotics or a thrombolytic agent for patients on lecanemab as cases of intracerebral hemorrhage of more than 1cm have been observed.

Namita A. Goyal, MD, a principal investigator on the pivotal phase 3 trial of NurOwn, talked about the importance of the upcoming FDA AdComm meeting and its impact on the ALS community.

In recognition of International Pompe Day, held on April 15, 2023, get caught up on some of the latest news in Pompe disease as the NeurologyLive® team shares some of our data updates.