Rare Neurological Diseases

Self-Administered Nanobody for Myasthenia Gravis Shows Phase 3 Efficacy

Sonia Reyes
Neurology & Mental Health, Rare Neurological Diseases | Apr 23, 2026
Gefurulimab, a once-weekly self-administered subcutaneous nanobody for gMG, met phase 3 PREVAIL endpoints at Week 26, potentially shifting care away from IV infusion centers.

A Once-Daily Epilepsy Pill With No Titration Clears Phase 3

Sonia Reyes
Neurology & Mental Health, Rare Neurological Diseases, Population Health Management | Apr 23, 2026
Azetukalner, a once-daily oral antiseizure agent requiring no titration, reduced focal onset seizures by 53% in phase 3 X-TOLE2, with an NDA filing expected mid-2026

First Brain-Penetrating Enzyme Therapy Wins FDA Approval for Hunter Syndrome

Sonia Reyes
Neurology & Mental Health, Rare Neurological Diseases | Apr 4, 2026
Denali's Avlayah wins accelerated FDA approval as the first brain-penetrating enzyme replacement therapy for Hunter syndrome's cognitive decline.

Rare Neurological Diseases

Self-Administered Nanobody for Myasthenia Gravis Shows Phase 3 Efficacy

A Once-Daily Epilepsy Pill With No Titration Clears Phase 3

First Brain-Penetrating Enzyme Therapy Wins FDA Approval for Hunter Syndrome

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