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🎯 📈 New data from an ongoing phase 2a trial revealed that RAP-219, an investigational TARPγ8-specific AMPAR negative all...
09/10/2025

🎯 📈 New data from an ongoing phase 2a trial revealed that RAP-219, an investigational TARPγ8-specific AMPAR negative allosteric modulator, met its primary end point in reducing long episodes among patients with drug-resistant focal onset seizures over an 8-week period. NYU Langone Health

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Patients treated with RAP-219 had a 77.8% reduction in clinical seizures, with 24% achieving seizure freedom over 8 weeks, potentially supporting the agent’s advancement to phase 3 trials.

09/10/2025

Join our Live National Broadcast: RADICAVA ORS® (edaravone):
A Proven Treatment Built on Experience. Hear from expert
panelists as they discuss clinical research on the efficacy and
safety of RADICAVA ORS® and share insights into timely
diagnosis and treatment considerations for patients. Register now
to secure your spot: bit.ly/3V1GV2r

See full Prescribing Information: bit.ly/45MDd2V

📊🔬Interim results from the phase 3 ARGUS trial showed that investigational EPX-100 was safe and generally tolerated in p...
09/10/2025

📊🔬Interim results from the phase 3 ARGUS trial showed that investigational EPX-100 was safe and generally tolerated in patients with , further supporting the agent’s development in this patient population. Dravet Syndrome Foundation 🧠⚡

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Phase 3 trial results reveal EPX-100's safety and tolerability in Dravet syndrome, supporting its potential as an effective adjunctive therapy.

Missed our latest Mind Moments? 🎙️Barry Byrne, MD, explains how new   gene therapy guidelines will:➡️ Standardize patien...
09/10/2025

Missed our latest Mind Moments? 🎙️
Barry Byrne, MD, explains how new gene therapy guidelines will:
➡️ Standardize patient monitoring
➡️ Expand care team collaboration
➡️ Address equity & access issues
Tune in here:
https://hubs.li/Q03Hq8k-0

🔷 New Series Launch: Tackling Gaps in LGS CareIn our newest roundtable, Scott Perry, MD, and Gita Gupta, MS, MS, reflect...
09/09/2025

🔷 New Series Launch: Tackling Gaps in LGS Care
In our newest roundtable, Scott Perry, MD, and Gita Gupta, MS, MS, reflect on the 2025 LGS Research Meeting of the Minds and spotlight practical solutions for care gaps in Lennox-Gastaut syndrome.

🎯 Topics include:
• Improving individualized treatment strategies
• Expanding caregiver education
• Designing smarter clinical trials
• Promoting stakeholder collaboration

▶️ Watch the full conversation now:
https://hubs.li/Q03Hq32K0

ICYMI:   expands fremanezumab (Ajovy) for kids 6–17 with migraine 🎯Dr. Patricia Pozo-Rosich breaks down the pediatric mi...
09/09/2025

ICYMI: expands fremanezumab (Ajovy) for kids 6–17 with migraine 🎯

Dr. Patricia Pozo-Rosich breaks down the pediatric migraine evolution—from sleep hygiene to CGRP to what’s next👇

The head of the neurology department at Vall d’Hebron University Hospital provided perspective on how migraine treatment might advance in the near future, highlighting emerging pathways like PACAP antagonists and long-acting toxins. [WATCH TIME: 3 minutes]

🧪 Personalized EID may reduce JCV risk in natalizumab-treated MS patientsA new study in RRMS found that lower natalizuma...
09/09/2025

🧪 Personalized EID may reduce JCV risk in natalizumab-treated MS patients
A new study in RRMS found that lower natalizumab trough concentrations (

A recent study reveals that personalized extended interval dosing of natalizumab significantly reduces JCV seroconversion risk in patients with MS.

Dianthus Therapeutics announced positive phase 2 data for its complement-targeting agent, claseprubart, in generalized m...
09/09/2025

Dianthus Therapeutics announced positive phase 2 data for its complement-targeting agent, claseprubart, in generalized myasthenia gravis. The investigational therapy showed statistically significant improvements in multiple efficacy measures. A phase 3 trial is now in the works following an upcoming FDA meeting.

New trial data reveals claseprubart shows significant efficacy in treating generalized myasthenia gravis, paving the way for potential FDA approval.

Bio-Thera Solutions has officially closed enrollment for its pivotal phase 2/3 trial of BAT4406F, a fully humanized anti...
09/08/2025

Bio-Thera Solutions has officially closed enrollment for its pivotal phase 2/3 trial of BAT4406F, a fully humanized anti-CD20 monoclonal antibody, following a positive IDMC recommendation.

🔍 What’s new:
– Statistically significant efficacy
– Strong safety profile
– Preps now underway for regulatory submission to China’s National Medical Products Administration.

Bio-Thera Solutions announced that its pivotal trial of BAT4406F, an anti-CD20 monoclonal antibody, for neuromyelitis optica spectrum disorder will close enrollment early after achieving statistically significant efficacy.

As gene therapy moves into broader clinical use for Duchenne muscular dystrophy (DMD), new consensus guidelines provide ...
09/08/2025

As gene therapy moves into broader clinical use for Duchenne muscular dystrophy (DMD), new consensus guidelines provide a roadmap for safe and equitable care.

On Mind Moments, Barry Byrne, MD, highlights:
• Practical considerations for clinicians delivering gene therapy
• Multidisciplinary monitoring strategies for immune-related events
• Addressing barriers in access, including financial and geographic constraints
• How these guidelines will shape the next wave of gene therapy trials

🎧 Listen now: https://hubs.li/Q03Hdhx30

A newly published study shows that MS patients using extended interval dosing (EID) with lower natalizumab trough levels...
09/08/2025

A newly published study shows that MS patients using extended interval dosing (EID) with lower natalizumab trough levels had lower rates of JCV seroconversion—a crucial factor in minimizing PML risk.

This personalized approach may not only extend safety but also help clinicians manage long-term therapy with more precision. 📈

A recent study reveals that personalized extended interval dosing of natalizumab significantly reduces JCV seroconversion risk in patients with MS.

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