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...
by CheckRare Staff | Nov 29, 2023
Richard Lewis, MD, Department of Neurology at Cedars-Sinai Medical Center, discusses diagnosing CIDP as well as the revised guidelines used to manage this rare disease. Transcription: There have been guidelines created over the years. Because it’s...
by CheckRare Staff | Nov 28, 2023
Christine Eng, MD, Chief Medical Officer at Baylor Genetics, discusses whole genome sequencing and other forms of genetic testing in diagnosing rare diseases. Transcription: All of the testing that we do really is designed to address unmet patient...
