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 17, 2023
Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses treating pediatric growth hormone deficiency (PGHD). Transcription: Most of the patients with pediatric growth hormone deficiency or PGHD, have a moderate form of the disease. They...
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 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...