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...
by CheckRare Staff | Dec 1, 2023
Andrew Bomback, MD, Columbia University Medical Center, gives an IC-MPGN and C3G overview, two rare inflammatory kidney diseases. Transcription: My name is Andrew Bomback. I am a nephrologist at Columbia University Irving Medical Center, where I...
by CheckRare Staff | Nov 30, 2023
TSHA-102 is an investigational gene transfer therapy presented by Taysha Gene Therapies to treat Rett Syndrome. This therapy utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate levels of MECP2 in the central nervous...
