by CheckRare Staff | Nov 15, 2023
Pam Vig, PhD, Head of Research and Development at Mirum Pharmaceuticals, discusses Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). Transcription: I’m Pam Vig, head of Research and Development at Mirum...
by CheckRare Staff | Nov 14, 2023
Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses the company’s focus on finding a novel treatment for pediatric growth hormone deficiency. Transcription: My name is Rick Hawkins, and I’m the CEO and Chairman of Lumos Pharma....
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...
