Phase 2 NOBLE Study

Andrew Bomback, MD, Columbia University Medical Center, discusses the Phase 2 NOBLE study, testing pegcetacoplan in patients with immune-complex MPGN or C3 glomerulopathy.       Transcription:  The NOBLE study is a really interesting study because what...

AAV Gene Therapy

Angelman Syndrome (AS) is a rare neurogenetic disorder that affects approximately 1 in 15,000 births worldwide. It is characterized by developmental delay, lack of verbal speech, seizures, sleep disorders, and motor impairments. Despite its significant impact on...

CIDP and Efgartigimod

Richard Lewis, MD, Department of Neurology, Cedars-Sinai Medical Center, explains CIDP and efgartigimod, a new treatment that targets FcRn.       Transcription: My name is Richard Lewis. I’m a Professor of Neurology at Cedar-Sinai Medical Center...

GTX-104 STRIVE-On Trial

The pivotal Phase 3 GTX-104 STRIVE-ON trial is a crucial step in evaluating the safety and efficacy for aSAH patients.     GTX-104 is a clinical-stage, injectable formulation of nimodipine. It is specifically designed to address the significant unmet medical...

Myasthenia Gravis Patient Inequalities

Gordon Smith, MD, Professor and Chair of Neurology, Virginia Commonwealth University, explains how the current healthcare system leads to inequalities in diagnosis and treatment of myasthenia gravis.     Transcription: I’m Gordon Smith. I’m a...