by CheckRare Staff | Dec 13, 2023
Acanthamoeba keratitis (AK) is a rare, sight-threatening eye infection that affects the cornea. It can cause extreme pain and light sensitivity. If left untreated, it can lead to severe vision loss or even blindness. However, a new drug candidate has shown promising...
by CheckRare Staff | Dec 12, 2023
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...
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...
