by CheckRare Staff | Dec 11, 2023
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...
by CheckRare Staff | Dec 7, 2023
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...
by CheckRare Staff | Dec 6, 2023
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...
by CheckRare Staff | Dec 5, 2023
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...
by CheckRare Staff | Dec 4, 2023
The groundbreaking seladelpar clinical trial results have shed new light on the treatment of PBC. Seladelpar is a first-in-class oral, selective peroxisome proliferator-activated receptor (PPAR) delta agonist, or “delpar”. It has shown the...
