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Neurology News Network. for the week ending December 21, 2024. [WATCH TIME: 4 minutes]

FDA Places Hold on CONNECT2-EDO51, Sevasemten Meets End Point in Becker, Tolebrutinib Gets Breakthrough Therapy Designation

Articles

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending December 20, 2024. 

NeurologyLive® Friday 5 — December 20, 2024

Managing ALS, particularly its respiratory complications, requires a collaborative multidisciplinary team—including neurologists, pulmonologists, and respiratory therapists—working together to address the complexities of respiratory failure, the leading cause of death in these patients.

Breathing Life Into ALS Care: The Vital Role of Multidisciplinary Teams

Although the primary goal was not met, prasinezumab demonstrated encouraging effects on secondary and exploratory measures, such as time to worsening motor function, Clinical Global Impression of Change, MDS-UPDRS motor scores.

Parkinson Agent Prasinezumab Misses Primary End Point But Shows Positive Effects in Other Outcomes

Assistant professor of neurosurgery and neuroscience at Mayo Clinic Arizona

The assistant professor of neurosurgery and neuroscience at Mayo Clinic Arizona discussed an ongoing early-stage study assessing the therapeutic potential of NRTX-1001 nerve cell therapy in drug-resistant unilateral mesial temporal lobe epilepsy. [WATCH TIME: 5 minutes]

Studying Nerve Cell Therapy NRTX-1001 to Treat Mesial Temporal Lobe Epilepsy: Jonathan Parker, MD, PhD

, has been with the team since September 2022. Follow her on Twitter 

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

FDA Grants MDA Kickstart Program Rare Pediatric Disease Designation and Orphan Drug Designation in Congenital Myasthenic Syndrome

IJMSC Insights offers a closer look at the latest research and the people behind it from the community of the International Journal of Multiple Sclerosis Care (IJMSC) and the Consortium of Multiple Sclerosis Centers (CMSC).

IJMSC Insights: Blanca De Dios Perez on Employment and Multiple Sclerosis

Pediatric epilepsy specialist at University of California, San Francisco.

At AES 2024, a duo of pediatric epilepsy specialists discussed how implementation science, validated blood biomarkers, and cross-disciplinary collaboration are advancing epilepsy care and closing research gaps. [WATCH TIME: 3 minutes]

Advancing Epilepsy Research Through Implementation Science and Biomarkers: Adam Numis, MD; Laura Kirkpatrick, MD

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats.

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Expert Insights on Novel NMOSD Treatments

The Evolving Understanding of NMOSD Pathophysiology and the Role of Complement System

Video content above is prompted by the following:

How has our understanding of the pathophysiology of NMOSD evolved over the years (Contentti 2021)?

What is the role of the complement system in the pathophysiology of NMOSD?

Three medical experts explore key insights on novel treatments for NMOSD, focusing on relapse management, disability accumulation, and the efficacy of new versus traditional therapies.

This discussion covers the factors leading to relapses and recurring attacks in NMOSD, how disease severity and relapses contribute to disability accumulation, and their impact on patients' quality of life.

Relapses and Disability Accumulation in NMOSD

This discussion examines how disability accumulation may be influenced by the use of less efficacious treatments, compares the efficacy of novel and traditional NMOSD agents in clinical practice, and reviews relapse rates associated with FDA-approved treatments versus off-label and older therapies.

Treatments With Higher Efficacy and Relapses in NMOSD

This discussion examines the 138-week data on ravulizumab for NMOSD, focusing on long-term relapse rates, and share practical tips for effective NMOSD management in clinical settings.

Ravulizumab Long-Term Data and Management of NMOSD

Jacqueline Nicholas, MD, MPH, discusses the evolution of NMOSD pathophysiology understanding and the critical role of the complement system in its development.

Jacqueline Nicholas, MD, MPH, discusses available treatment options for NMOSD, including management of acute phases, relapses, and symptom control, as well as how both traditional and newer therapies address different aspects of NMOSD pathophysiology.

Treatment Options to Manage and Prevent Relapses in NMOSD

Jacqueline Nicholas, MD MPH discusses the importance of targeting NMOSD through different mechanisms of action and highlight key points for communicating with neurologists about the role of the complement system in the disease.

Importance of Diverse Mechanisms of Action to Effectively Treat NMOSD

Sean Pittock, MD, discusses how complement inhibitors are positioned in the treatment landscape of neuromyelitis optica spectrum disorder, providing an overview of ravulizumab, the most recent FDA-approved treatment, including its development rationale, half-life, dosing regimen, and new signals from the recently presented 138-week long-term extension data at EAN 2024.



Complement Inhibitors in NMOSD

Sean Pittock, MD, discusses the risks of meningococcal infection associated with eculizumab and ravulizumab in phase 3 and long-term extension studies, as well as his interpretation of the concerns surrounding meningococcal vaccination and its potential impact on relapse activity.


Meningococcal Infection and NMOSD

Sean Pittock, MD, discusses the long-term efficacy and safety results of other FDA-approved treatments for neuromyelitis optica spectrum disorder, including satralizumab, inebilizumab, and the off-label use of rituximab.

Long-term NMOSD Data

Sean Pittock, MD, discusses how he utilizes long-term data in his clinical practice for treating neuromyelitis optica spectrum disorder and offers advice for physicians on incorporating these insights into patient care.

Use of Long-term NMOSD Data in Clinical Practice

 video series brings together pairs of expert clinicians and physicians to independently provide their insights on the latest and most up-to-date clinical care for specific neurologic diseases and disorders.

The Evolving Understanding of NMOSD Pathophysiology and the Role of Complement System

EP: 1.Relapses and Disability Accumulation in NMOSD

EP: 2.Treatments With Higher Efficacy and Relapses in NMOSD

EP: 3.Ravulizumab Long-Term Data and Management of NMOSD

EP: 4.The Evolving Understanding of NMOSD Pathophysiology and the Role of Complement System

EP: 5.Treatment Options to Manage and Prevent Relapses in NMOSD

EP: 6.Importance of Diverse Mechanisms of Action to Effectively Treat NMOSD

EP: 7.Complement Inhibitors in NMOSD

EP: 8.Meningococcal Infection and NMOSD

EP: 9.Long-term NMOSD Data

EP: 10.Use of Long-term NMOSD Data in Clinical Practice