by CheckRare Staff | Nov 8, 2023
CymaBay Therapeutics, Inc., has initiated a Phase 3b/4 study called AFFIRM to evaluate the effect of seladelpar on clinical outcomes in patients with cirrhosis due to PBC. Image Source: Dreamstime Primary Biliary Cholangitis (PBC) is a rare, chronic...
by CheckRare Staff | Nov 8, 2023
DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with DM1[^2^]. Image: Dreamstime This therapy consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense...
by CheckRare Staff | Nov 6, 2023
Joseph Mikhael, MD, Chief Medical Officer at the International Myeloma Foundation explains racial disparities in the diagnosis of Multiple Myeloma Transcription: They don’t have the same incidents level that we see in the African population, but...
by CheckRare Staff | Nov 2, 2023
Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement for EveryLife Foundation for Rare Diseases, explains how new technologies in newborn screening aren’t being taken advantage of due to their costs. Transcription: The Everylife...
by CheckRare Staff | Nov 1, 2023
Monica Gadelha, PhD, Professor of Medicine at the University of Rio De Janeiro discusses the use of paltusotine in patients with acromegaly. Transcription: GH is secreted, controlled by somatostatin. Somatostatin is a molecule that produces the...
