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NeurologyLive® Friday 5 — September 29, 2023

The AI-based SOMNUM technology uses diagnostic algorithms based on multi-channel/time series sleep biosignal data to deliver fast, accurate diagnostic information for providers.

NeurologyLive® Staff

Mechanistic Approach Behind AMX0035 in Progressive Supranuclear Palsy: Gunter Hoglinger, MD

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 29, 2023.

Gunter Hoglinger, MD

FDA Approves Cipaglucosidase Alfa and Miglustat as First Two-Component Therapy for Pompe Disease

The professor of neurology and translational neuroscientist at Ludwig-Maximillian’s University Munich detailed the logic behind the ORION study, a new trial assessing AMX0035, an approved therapy for ALS, in patients with progressive supranuclear palsy. [WATCH TIME: 4 minutes]

Recent Advances in Epilepsy Management, New Treatments and Innovative Procedures: Patricia C. Dugan, MD

In a phase 3 setting, the 2-component therapy showed positive trends or clinically meaningful improvements on motor and respiratory functions compared with previously approved enzyme replacement therapy.

Patricia C. Dugan, MD

Clinical Insights on the Emerging Trends in Research for NMOSD and MOGAD

The associate professor of neurology at NYU Grossman School of Medicine discussed her talk at IFN 2023 on advancements in epilepsy treatments, highlighting new medications and surgical options. [WATCH TIME: 3 minutes]

PACAP-Targeted Antibody Lu AG09222 Demonstrates Positive Results in Phase 2 Migraine Trial

Erin Longbrake, MD, PhD, associate professor of neurology at Yale School of Medicine, discussed the importance of understanding rare autoimmune disorders to effectively design clinical trials and treatment strategies, especially for heterogeneous conditions like NMOSD and MOGAD.

Adjusting Motor Fluctuation Management in an Era of Neuromodulation: Laxman Bahroo, DO

Over a 4-week treatment period, patients in both Lu AG09222 groups experienced about 2 fewer migraine days per month compared with placebo.

Laxman Bahroo, DO

NeurologyLive® Clinician of the Month Spotlight: Peter B. Kang, MD, FAAN, FAAP

The professor of neurology and resident program director at MedStar Georgetown University Hospital discussed how Parkinson disease motor fluctuations are managed amid new, novel technologies incorporated in the field. [WATCH TIME: 4 minutes]

Empowering Women to Develop Leadership Skills in Neurology: Lara V. Marcuse, MD

As part of our monthly clinician spotlight, NeurologyLive® highlighted neueomuscular expert Peter Kang, MD, director of the Paul and Sheila Wellstone Muscular Dystrophy Center and professor of neurology at the University of Minnesota.

Lara V. Marcuse, MD

Disparities in Memory Clinic Visits and Dementia Severity Identified in Disadvantaged Neighborhoods

The codirector of the Mount Sinai Epilepsy Program at the Icahn School of Medicine at Mount Sinai talked about the Women in Neurology and Leadership session she spoke in at IFN 2023. [WATCH TIME: 3 minutes]

FDA Advisory Committee Downvotes Mesenchymal Stem Cell Approach NurOwn as Potential ALS Therapy

Patients who self-identified as Black and were living in less affluent neighborhoods were less likely to be seen in one large memory clinic as well as have more severe dementia at initial visit.

Tau-Targeting Agent ADEL-Y01 Gains FDA Clearance for Phase 1 Study

After deliberating, the FDA committee expressed concerns about the mechanism of action of NurOwn, manufacturing details, and consistency of data.

Perfecting a Cocktail of Approaches to Treat Alzheimer Disease: Richard Isaacson, MD

By targeting tau-ack280, ADEL-Y01 inhibits tau aggregation and propagation, and promotes microglial tau clearance.

Richard Isaacson, MD

Axsome Therapeutics Postpones Data Readout for Phase 3 SYMPHONY Trial

The director of the Center for Brain Health and Alzheimer Prevention Clinic at Florida Atlantic University provided perspectives on the multimodal, multitargeted approach needed to treat Alzheimer disease. [WATCH TIME: 5 minutes]

Clinical Perspective on the Advances and Challenges in Multiple Sclerosis Treatment: Patricia K. Coyle, MD

The phase 3 SYMPHONY trial assessing AXS-12, a selective norepinephrine reuptake inhibitor for narcolepsy, was initially expected in the third quarter of 2023.

Patricia K. Coyle, MD

NeuroVoices: Craig Blackstone, MD, PhD, FANA, on Helping Path Research Careers for Young Medical Professionals

The professor of neurology at Stony Brook University Medical Center discussed the progress observed in the field of multiple sclerosis and the lack of effective treatments for progressive forms of the disease. [WATCH TIME: 3 minutes]

Coya Therapeutics Announces Plans for FDA Discussion on ALS Agent COYA 302

The chief of the Movement Disorders Division at Mass General Hospital provided context on the goals behind the research careers reimagined course at the recently concluded ANA Annual Meeting.

Standard and Nonstandard Physical Therapy Show Similar Effectiveness for Parkinson Disease

Coya Therapeutics expects to release data from the biomarker analyses of COYA 302 and publish the full results from the trial in a peer-reviewed journal in the second half of the year.

Advancements in Stroke Care, Ischemic Stroke, and Cerebral Hemorrhages: Matthew Schrag, MD, PhD

Although research supports the use of physical therapy as an effective treatment for Parkinson disease, implementation factors including type, timing, frequency, and durability of outcomes remain mainly untouched.

Matthew Schrag, MD, PhD

Declined Odor Sensitivity Observed in APOE ε4 Carriers

The associate professor of neurology at Vanderbilt University Medical Center discussed the progress in stroke care, focusing on ischemic strokes and cerebral hemorrhages, and their connection with vascular diseases and cognitive disorders. [WATCH TIME: 3 minutes]

The Complex Landscape of NMOSD and MOGAD-Associated Diseases: Erin Longbrake, MD, PhD

Between ages 65 to 69, gene carriers of APOE ε4 could detect an average of about 3.2 of the smells presented, compared with about 3.9 smells for noncarriers of the gene variant.

Erin Longbrake, MD, PhD

New Interim Data Reported on DNL310 for Hunter Syndrome Treatment in Phase 1/2 Trial

The associate professor of neurology at Yale School of Medicine discussed the differentiation of neuromyelitis optica spectrum disorders and myelin oligodendrocyte glycoprotein -associated disease from multiple sclerosis at IFN 2023. [WATCH TIME: 5 minutes]

IJMSC September/October 2023 Highlights

Additional biomarker findings from up to 2 years of DNL310 treatment suggest positive changes in adaptive behavior and cognition as well as improvement in auditory function among pediatric patients with Hunter syndrome.

Erin E Gyomber

CNM-Au8 Shows Significant Long-Term Survival Improvement in HEALEY ALS Platform Trial

Slideshows

Review the latest peer-reviewed articles dedicated to the multidisciplinary management of multiple sclerosis published in the International Journal of MS Care.

Challenges With Access, Delivery of New ALS Therapeutics: Richard Bedlack, MD, PhD, MS

In an integrated meta-analysis of the HEALEY ALS platform trial and RESCUE-ALS trial, 30mg of CNM-Au8 resulted in 59% decreased risk of mortality among participants with ALS compared with PRO-ACT matched placebo over long-term follow-up.

Richard S. Bedlack, MD, PhD, MS

Role of Gut Microbiome, Alpha Synuclein in the Prodromal Stage of Parkinson Disease

The director of the Duke ALS Clinic at Duke Health provided perspective on the issues with getting newly approved ALS therapies to patients who need them the most. [WATCH TIME: 4 minutes]

Promise in PACAP Pathway for Treating Migraine: Jessica Ailani, MD

Virginia Gao, MD, PhD, a movement disorders fellow at Weill Cornell Medicine, discussed several topics related to the use of biomarkers in the prodromal stages of neurodegenerative disorders like Parkinson disease.

Jessica Ailani, MD