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NeurologyLive®
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Delivering healthcare professionals treating neurological diseases quality and relevant information to help achieve the best patient care possible. #MindMoments

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🧬 AskBio’s #genetherapy AB-1009 for late-onset Pompe disease has received #FDA IND clearance, plus Fast Track & Orphan Drug designations.

🔗 ➡️ www.neurologylive.com/view/askbio-...
#RareDisease #CGT #Neurology #GeneTherapy #ClinicalTrials
AskBio’s Late-Onset Pompe Disease Gene Therapy AB-1009 Cleared for Phase 1/2 Trial | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
AskBio noted that AB-1009 also recently received fast track and orphan drug designations from the FDA.
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January 23, 2026 at 3:11 PM
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🧠💬 Mood & behavioral symptoms are core features of #Parkinsondisease

Sneha Mantri, MD, MS, discusses why depression & anxiety often emerge early, how they shape quality of life, and how clinicians can better screen and treat them.

Read more 👇
www.neurologylive.com/view/neurovo...
#Neurology
NeuroVoices: Sneha Mantri, MD, MS, on Addressing Depression and Anxiety in Parkinson Care | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
The chief medical officer at the Parkinson’s Foundation detailed how mood and behavioral symptoms shape quality of life in Parkinson disease and how clinicians can better identify and manage them.
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January 22, 2026 at 2:50 PM
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🎥 New Roundtable 🔽

Investigating Depressive Symptoms in Youth with Refractory Epilepsy. Experts unpack key findings, assessment gaps, and clinical implications across a 4-part series 👧⚡️

Watch now 👉 www.neurologylive.com/roundtable-d...
#Epilepsy #MentalHealth #Depression #ChildHealth #Neurology
January 21, 2026 at 2:06 PM
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🧬 EC approves high-dose Spinraza for #SMA 🇪🇺
Backed by DEVOTE & RESPOND data, the new nusinersen regimen delivers higher exposure earlier, supporting stronger motor neuron preservation and meaningful clinical gains 💙

Read more 👉 www.neurologylive.com/view/reactio...
#Spinraza #Neurology
What the EC Decision on High-Dose Spinraza Means for SMA Care | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Stephanie Fradette, PharmD, head of neuromuscular development at Biogen, gave immediate reaction to the EC approval of high-dose nusinersen and its implications for evolving SMA treatment strategies.
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January 20, 2026 at 2:13 PM
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🧠💉 #FTD research update
Investigational COYA 302 showed stable cognition over 6 months in a small FTD trial, alongside rapid & sustained Treg enhancement. FDA has now accepted the IND. Early but promising

🔗 ➡️ www.neurologylive.com/view/investi...
#Neurology #ClinicalTrials #BiotechNews #FDA
Investigational COYA 302 Shows Feasibility, Preliminary Efficacy in Small Trial of Frontotemporal Dementia | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Coya Therapeutics reveals promising results for COYA 302, a potential treatment for frontotemporal dementia, showing cognitive stability and Treg enhancement.
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January 19, 2026 at 2:10 PM
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🧬 Newborn Screening for #Duchenne (DMD) is here❗
DMD & MLD were added to the US RUSP—unlocking earlier diagnosis, earlier treatment, and stronger family support 🍼💪

Read more: www.neurologylive.com/view/newborn...
#RareDisease #Neurology #NewbornScreening #Neuromuscular #PublicHealth #GeneticTesting
Newborn Screening for Duchenne: Clinical and Policy Implications | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Paul Melmeyer, PhD, executive vice president of public policy and advocacy at the Muscular Dystrophy Association, explains how adding Duchenne muscular dystrophy to the Recommended Unified Screening P...
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January 16, 2026 at 2:03 PM
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🧬 Promising DMD data from #REGENXBIO: RGX-202 patients beat expected disease progression on NSAA at 12 & 18 months. 🚀 BLA planned for 2026 as trials expand globally.

Read more: www.neurologylive.com/view/patient...
#DMD #GeneTherapy #ClinicalTrials #RareDisease #Neurology #BrainHealth
Patients Treated With REGENXBIO’s DMD Gene Therapy RGX-202 Exceed Expected Disease Trajectory on NSAA | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
In the middle of 2026, the company is planning to submit a BLA for RGX-202 via an accelerated approval pathway.
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January 15, 2026 at 2:07 PM
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🧬 #FDA approves Zycubo—the FIRST treatment for pediatric Menkes disease!

💉 Subcutaneous copper histidinate boosts survival, especially when started in the first 4 weeks of life. What could newborn screening change? 👶✨

Read more: www.neurologylive.com/view/fda-app...
#MenkesDisease #FDAApproval
FDA Approves Subcutaneous Copper Histidinate as First Treatment for Pediatric Menkes Disease | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
The FDA approved Sentynl Therapeutics’ copper histidinate for the treatment of pediatric Menkes disease, a rare genetic neurodegenerative disorder, following an earlier complete response letter.
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January 14, 2026 at 2:01 PM
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🧠✨ Promising news in Dravet syndrome: Phase 3 EMPEROR results for zorevunersen are now expected by mid-2027, with an FDA NDA planned soon after. A potential step toward disease modification.

Read more: www.neurologylive.com/view/phase-3...
#Dravet #EpilepsyResearch #RareDisease #Biotech #Neurology
Phase 3 Data Readout of EMPEROR Study to Come Earlier, With FDA Submission Expected to Follow | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Those from the clinical community may expect to hear data from the registrational trial assessing zorevunersen in Dravet syndrome by mid-2027.
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January 13, 2026 at 2:25 PM
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Big news in neurology❗
Annexon has submitted an EMA marketing application for tanruprubart, a first-in-class C1q antibody for Guillain-Barré syndrome.

#Phase3 data show faster, more complete recovery & QoL gains. Read more below 👇
www.neurologylive.com/view/annexon...
#GBS #Neurology #Biotech
Annexon Submits Marketing Authorization Application for Tanruprubart to Treat Guillain-Barré Syndrome | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Annexon submitted an EMA marketing application for tanruprubart, a first-in-class C1q monoclonal antibody that showed faster and more complete recovery in Guillain-Barré syndrome patients across rando...
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January 12, 2026 at 2:12 PM
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🧠⚡ BMB-101 advances toward registrational trials after positive phase 2 data in drug-resistant absence seizures & developmental epileptic encephalopathies.

Read more on what’s next: www.neurologylive.com/view/bmb-101...
#Epilepsy #ClinicalTrials #NeuroPharma #RareDisease #DrugDevelopment #Seizures
BMB-101 Heads for Registrational Trials Following Positive Phase 2 Data in Absence Seizures, Developmental Encephalopathies | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Bright Mind Biosciences reveals promising results for BMB-101 in treating drug-resistant seizures, paving the way for future clinical trials.
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January 9, 2026 at 2:12 PM
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🧠💊 New #epilepsy research: Fenfluramine improved everyday executive functioning in adults with Lennox-Gastaut syndrome—independent of seizure reduction.

Read more 👉 www.neurologylive.com/view/fenflur...
#LGS #Neurology #AES2025 #MedicalNews #BrainHealth
Fenfluramine Treatment Leads to Long-Term Improved Everyday Executive Functioning in Lennox-Gastaut Syndrome | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
A recent analysis reveals fenfluramine's positive effects on executive functioning in adults with Lennox-Gastaut syndrome, independent of seizure reduction.
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January 8, 2026 at 2:21 PM
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🧠🩺 Early treatment can change lives—but inequities still delay care for infants with epileptic spasms. At #AES2025, Drs. Christina Briscoe & Emily Spelbrink unpacked system-level barriers and paths toward equity

Read more 👇
www.neurologylive.com/view/neurovo...

#Epilepsy #Neurology #PedsNeuro
NeuroVoices: Christina Briscoe, MD; Emily Spelbrink, MD, PhD, on Addressing Equity Gaps in Infantile Epileptic Spasms Syndrome | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
A pair of pediatric epileptologists highlight the systemic and patient level factors driving inequities in infantile epileptic spasms syndrome care and outline paths toward more equitable diagnosis an...
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January 7, 2026 at 2:08 PM
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🧠📊 #FDA Action Update: December 2025
From a low-sodium oxybate NDA acceptance to a new approval in gMG —and key trial updates in ALS & MS—the FDA closed out 2025 with major neurology decisions shaping the pipeline

Read more 👉 www.neurologylive.com/view/fda-act...
#Neurology #ClinicalTrials #Approve
FDA Action Update, December 2025: Acceptance, Approval, and Clearance | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Catch up on any of the neurology news headlines you may have missed in December 2025, compiled into 1 place by the NeurologyLive® team.
www.neurologylive.com
January 6, 2026 at 2:56 PM
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🧠 New 2025 #NMOSD diagnostic guidelines are here!

Experts clarify AQP4+ vs antibody-negative disease, spotlight mimics like MOGAD, and aim to protect access to FDA-approved therapies 💊

Read more → www.neurologylive.com/view/clinica...
#Neurology #RareDisease #ClinicalGuidelines
A Clinical Review of the 2025 Neuromyelitis Optica Spectrum Disorder Guidelines | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Benjamin Greenberg, MD, MHS, a nationally recognized neuroimmunologist and contributor to the NMOSD diagnostic guidelines, spoke about the clinical impact of the recently published recommendations and...
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January 5, 2026 at 2:21 PM
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🧠 NeurologyLive® Year in Review 2025 highlights key #Alzheimer disease trials that could reshape care—spanning anti-amyloid, anti-tau, immunotherapy, and prevention-focused studies

From DIAN-TU to AHEAD 3-45, here’s what to watch 👇 www.neurologylive.com/view/neurolo...
#Neuro #ADResearch #AD
NeurologyLive® Year in Review 2025: What Trials Could Reshape Alzheimer Care? | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
As part of NeurologyLive®'s Year in Review, take a look at some of the top ongoing clinical trials in Alzheimer disease and dementia that may impact care.
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January 2, 2026 at 2:13 PM
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At #AES2025, Joseph Sullivan, MD reviewed early POLARIS phase 1/2 data for ETX101, a one-time AAV9 gene regulation therapy targeting SCN1A in inhibitory interneurons—showing encouraging #seizureReduction & developmental gains in young children.

👉 Read more: www.neurologylive.com/view/neurovo...
NeuroVoices: Joseph Sullivan, MD, on ETX101 Gene Regulation Therapy and Early POLARIS Findings in Dravet Syndrome | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
The director of the Pediatric Epilepsy Center at UCSF reviewed recently presented interim phase 1/2 data for ETX101, a one-time AAV9 gene regulation therapy aiming to increase SCN1A expression in inhi...
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December 24, 2025 at 2:41 PM
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🧠🎮 Test your neurology knowledge!
This week’s NeurologyLive® Brain Games dives into neuromodulation for Parkinson disease—covering DBS targets, adaptive systems & more. Take the 3-question quiz and share your score!

Read more 👉 www.neurologylive.com/view/neurolo...
#Neurology #PD #Braingames
NeurologyLive® Brain Games: December 21, 2025 | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on neuromodulation for Parkin...
www.neurologylive.com
December 23, 2025 at 2:07 PM
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🧠 Breakthrough in #Parkinson research 🔬

GT-02287 reduced CSF GluSph, signaling improved GCase activity, with favorable safety in Phase 1b trials. A key biomarker milestone for #PD therapies.

Read more 👉 www.neurologylive.com/view/brain-p...
#Biotech #ClinicalTrials #Neurology #Neuroscience
Brain-Penetrant Molecule GT-02287 Demonstrates Reversal of Glucosylsphinogosine in Parkinson Disease | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Gain Therapeutics advances Parkinson's treatment with GT 2287, showcasing promising safety and tolerability in ongoing trials. Discover the latest findings.
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December 22, 2025 at 2:19 PM
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🚨 New Podcast Launch 🚨

Frontlines with Frontera makes its debut today! Hosted by Jennifer Frontera, MD, the series brings thoughtful, clinician-to-clinician conversations on the toughest questions in acute #neurology and #neurocritical care.

Episode 1 is live! www.neurologylive.com/view/frontli...
Frontlines with Frontera: Using Artificial Intelligence to Rethink Seizure Management in the Neuro ICU | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Frontlines with Frontera, a podcast hosted by neurointensivist Jennifer Frontera, MD, brings you an exclusive interview with Sahar Zafar, MD, MBBS. [LISTEN TIME: 30 minutes]
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December 19, 2025 at 7:41 PM
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🧬 Genetic #Epilepsy in Adults Network (GEAN) aims to standardize genetic testing in adults and define adult outcomes for rare genetic epilepsies. GEAN is a new AES-funded consortium and Elizabeth Gerard, MD explains why it matters.

🔗 ➡️ www.neurologylive.com/view/genetic...
#AES2025 #Genetic #Gene
Genetic Testing in Adult Epilepsy: GEAN’s Plan to Standardize Practice and Study Adult Outcomes | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Elizabeth Gerard, MD, an epileptologist at Northwestern Medicine, provided clinical insights on a new consortium, GEAN, which aims to expand genetic testing and define adult phenotypes for rare geneti...
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December 17, 2025 at 2:41 PM
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🧠⚡ Long-term data on azetukalner (XEN1101), a first-in-class Kv7 channel opener, show sustained #seizure reduction and meaningful seizure-freedom signals in focal onset seizures.

What could this mean for care? Read more 👉 www.neurologylive.com/view/underst...
#Epilepsy #AES2025 #Neurology
Understanding the Therapeutic Potential and Promising Early-Stage Data on Azetukalner for Focal Onset Seizures | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Christopher Kenney, MD, FAAN, chief medical officer at Xenon Pharmaceuticals, reviews encouraging long-term data behind azetukalner, a first-in-class Kv7 channel opener, as a potential treatment for f...
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December 16, 2025 at 2:22 PM
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Post–cardiac arrest seizures demand precision, not just more monitoring. At #AES2025, Andrea Rossetti, MD highlights why routine #EEG can rival continuous EEG, how prognostication guides treatment, and where broader-spectrum ASMs may fit best.

Read more: www.neurologylive.com/view/refinin...
Refining Prognosis and Treatment in Post–Resuscitation Epilepsy Care | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Andrea Rossetti, MD, director of the EEG/Epilepsy Unit at the Department of Clinical Neurosciences the Lausanne University Hospital in Switzerland, offers insight into post-resuscitation epilepsy care...
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December 15, 2025 at 2:48 PM
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🎧 New Mind Moments episode!
Sneha Mantri, MD, MS joins the podcast to break down how mood, anxiety, and behavior shape life with Parkinson disease—often long before motor symptoms appear.

Listen here ➡️ www.neurologylive.com/view/episode...
#PD #Neurology #MovementDisorders #MindMoments #NeuroCare
Episode 156: Building Better Mood and Behavior Care for Parkinson Disease | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sneha Mantri, MD, MS. [LISTEN TIME: 24 minutes]
www.neurologylive.com
December 12, 2025 at 2:04 PM
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🚨 Major #DMD breakthrough: Deramiocel hits its #Phase3 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: www.neurologylive.com/view/cell-th...

#RareDisease #Biotech #Neurology
Cell Therapy Deramiocel Meets Primary End Point in Phase 3 DMD Cardiomyopathy Trial | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
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.
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December 11, 2025 at 2:05 PM