by CheckRare Staff | Nov 13, 2023
This article explores the significance of this clearance, the potential benefits of KB408, and the path forward for addressing Alpha-1 Antitrypsin Deficiency. Image: Dreamstime In a significant development for the treatment of Alpha-1 Antitrypsin...
by CheckRare Staff | Nov 10, 2023
Alagille syndrome is a rare disease that typically presents in infants within the first three months of life.[1,2] Patients may present a wide spectrum of signs and symptoms.[1,2] The first recognized cases were reported in 1969 by Daniel Alagille in France.[3,4] It...
by CheckRare Staff | Nov 9, 2023
This article delves into the impact of setmelanotide on metabolic syndrome risk, the analyses from the ROAD genetic testing program, and the role of precision medicine in the treatment of MC4R pathway diseases. Image: Dreamstime In recent years, there...
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...
