NeurologyLive

12/12/2025

🧠 Stay up to date on the latest in ! The NeurologyLive® team has compiled all November 2025 U.S. Food and Drug Administration updates in 1 place — including approvals, new designations, submissions, resubmissions, and clinical trial movements.

✅ Catch up on the headlines you may have missed here: https://hubs.li/Q03XS8mQ0

12/12/2025

: The U.S. Food and Drug Administration has approved inebilizumab (Uplizna; Amgen for the treatment of generalized myasthenia gravis (gMG).

This marks the third FDA-approved indication for inebilizumab, which is also approved for antiaquaporin-4 antibody positive neuromyelitis optica spectrum disorder and immunoglobulin G4–related disease.

Read More: https://hubs.li/Q03X-nqX0

12/12/2025

🧠💬 New Patient Interview on Narcolepsy in Media!

In a conversation with Julie Flygare, JD, president and CEO of Project Sleep, she discussed how inaccurate media portrayals of may contribute to stigma, confusion about symptoms, and delays in clinical diagnosis.

Watch the full conversation to learn more about the impact of representation on patient experiences and clinical care: https://hubs.ly/Q03XRZ-g0

12/11/2025

At , Dr. Michael Sperling, MD, accepted the Founders Award and shared insights on the future of epilepsy. He highlighted how advances in monitoring, biomarkers, and clinical trials can push the field closer to individualized epilepsy care🔬

🔗 ➡️ https://hubs.li/Q03XX7j20

Michael Sperling, MD, Baldwin Keyes Professor of Neurology at Vice Chair for Research at the Sidney Kimmel Medical College, spoke about the significance of receiving the Founders Award and the current state of epilepsy.

12/11/2025

📱✨ New insights from highlight the power of mobile health tools in epilepsy care

A newly presented poster from the HOBSCOTCH-3 trial showed that the HOBSCOTCH mobile app is a feasible and effective way to collect daily self-reported outcomes from adults with epilepsy. Across 109 participants, more than 22,000 daily logs were submitted 📊🧠

While seizure frequency and medication adherence remained stable across phases, researchers identified meaningful trends in:
🔹 Memory strategy use
🔹 Emotional well-being
🔹 Day-to-day engagement
— especially during the active intervention and booster periods.

Lead author Robert Ross-Shannon, MS and colleagues emphasized that mobile platforms can offer real-time, high-resolution insights that traditional methods often miss. These patterns may be key to understanding how cognitive-behavioral interventions work in daily life. 💡

🗝️ takeaway: Mobile health tools like HOBSCOTCH show strong potential for personalized epilepsy care, improved self-management, and more responsive digital intervention models. 🚀

Read more: https://hubs.li/Q03XQzWG0

A new study presented at AES 2025 reported that a cognitive-behavioral intervention via mobile application for patients with epilepsy can reliably capture daily self-reported outcomes.

12/11/2025

Progress in 📈

🔹 60% median seizure reduction at 18–24 months
🔹 Consistent benefit across Dravet, LGS & other DEEs
🔹 Favorable long-term safety

Bexicaserin is now advancing through global Phase 3 trials.
Read more here ➡️ https://hubs.li/Q03XQvHk0

New findings reveal bexicaserin significantly reduces seizure frequency in patients with developmental and epileptic encephalopathies, supporting its advancement to phase 3 trials.

12/11/2025

🚨 Major breakthrough: Deramiocel hits its Phase 3 endpoint!

💪 54% slower loss of upper-limb function
❤️ 91% slower decline in cardiac function

A huge moment for Duchenne research and regulatory momentum. Read here: https://hubs.li/Q03XQnn50

Capricor Therapeutics anticipates that detailed phase 3 data from the HOPE-3 study will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.

12/11/2025

📊 AES 2025 findings: A 10-year review of MOGAD patients showed that refractory status epilepticus, characteristic EEG slowing, and cortical–deep MRI abnormalities may serve as early biomarkers of severe, refractory epilepsy.

14/49 patients had seizures, half presenting initially. The study held important insights for risk stratification. 🧠

A study reveals EEG and MRI biomarkers in MOGAD patients with epilepsy, highlighting potential early indicators for refractory epilepsy and improved treatment outcomes.

12/10/2025

👶🧠 New phase 2 data from the SP0968 trial show lacosamide reduced electrographic seizure burden in neonates with ENS and was well tolerated. PK findings also aligned closely with adult exposure levels.
Full details:

Findings from a phase 2 trial highlighted lacosamide's effectiveness and safety in treating neonatal seizures, showing significant seizure reduction and tolerability in patients.

12/10/2025

🔬 New phase 2 trial: stiripentol (Diacomit), approved for Dravet syndrome, showed reductions in seizure burden in Lennox-Gastaut syndrome across multiple seizure types.
Study used baseline → placebo → active treatment design over 120 days.
Encouraging data for a difficult-to-treat epilepsy.

A new phase 2 study presented at AES 2025 showed that stiripentol may reduce seizure burden in patients with Lennox-Gastaut syndrome.

12/10/2025

Regulatory update: The FDA has accepted the NDA for TRN-257, a controlled-release therapy targeting cataplexy and excessive daytime sleepiness in narcolepsy, as well as EDS in idiopathic hypersomnia.

The formulation uses Tris Pharma’s RaftWorks and LiquiXR platforms to deliver a once-nightly, low-sodium option—an important consideration for long-term management. PDUFA is set for June 20, 2026.

TRN-257, a treatment in development for adults with narcolepsy and idiopathic hypersomnia, is designed to reduce daily sodium exposure relative to existing higher-sodium therapies.

12/10/2025

🧠 AES 2025 Update: New interim data from the POLARIS program evaluating ETX101, an AAV9 gene replacement therapy for Dravet syndrome, demonstrated a favorable safety profile and dose-dependent reductions in seizure burden across 19 treated patients.

NeurologyLive interviewed principal investigator Joseph Sullivan, MD, who detailed early anti-seizure signals, developmental improvements at higher doses, and how these findings are shaping future dose decisions and trial design.

📲

The director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco detailed early phase data of Encoded Therapeutics’ ETX101 gene replacement therapy in children with Dravet syndrome. [WATCH TIME: 3 minutes]