by CheckRare Staff | Nov 27, 2023
Eric Crombez, MD, Chief Medical Officer of Ultragenyx, discusses the latest results from the Phase 2/3 Orbit Study targeting osteogenesis imperfecta. Transcription: Orbit is the name we have given to our ongoing phase 2/3 study. We have been talking...
by CheckRare Staff | Nov 24, 2023
Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses the OraGrowth212 Clinical Trial testing LUM-201 (ibutamoren) to treat pediatric growth hormone deficiency (PGHD). Transcription: We have a global trial up and running. There are 80...
by CheckRare Staff | Nov 23, 2023
Pam Vig, PhD, Head of Research and Development at Mirum Pharmaceuticals, highlights some of the clinical data on progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome presented at the North American Society For Pediatric Gastroenterology,...
by CheckRare Staff | Nov 22, 2023
Eric Crombez, MD, Chief Medical Officer of Ultragenyx, talks about treating and diagnosing osteogenesis imperfecta patients. Transcription: Patients definitely can have a very different path to diagnosis. This disease does have a spectrum of...
by CheckRare Staff | Nov 21, 2023
IND Application Approved For DM1 Treatment The FDA has approved the investigational new drug (IND) application, initiating the FREEDOM-DM1 Phase 1 study of PGN-EDODM1 in DM1 patients in the U.S. What is DM1? Myotonic dystrophy type 1 (AKA Steinert’s Disease) is a...
