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...
by CheckRare Staff | Nov 20, 2023
Christine Eng, MD, Chief Medical Officer at Baylor Genetics discusses the diagnostic lab and her involvement in the organization. Transcription: My name is Christine Eng. I am a pediatrician and a medical geneticist. I’ve been in this field for...
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 16, 2023
The FDA has given clearance for a pivotal gastric cancer Phase III trial. Osemitamab (TST001) offers hope for patients with HER2-negative gastric or gastroesophageal adenocarcinoma, a disease with limited treatment options and poor survival rates. By...
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...
