by CheckRare Staff | Feb 8, 2023
Gerald Vockley, MD, Ph.D., Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, provides a brief history of newborn screening. Newborn screening began in the 1960s with phenylketonuria (PKU), pioneered by Dr. Robert...
by CheckRare Staff | Feb 3, 2023
Shaji K. Kumar, MD, Professor of Medicine at Mayo Clinic, discusses long-term efficacy and post-hoc subgroup analyses of MAIA, a phase 3 study evaluating the efficacy of daratumumab in combination with lenalidomide and dexamethasone (D-Rd) to that of...
by CheckRare Staff | Feb 2, 2023
P.J. Brooks, Ph.D., Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the two main types of gene therapy currently in development: ex vivo and in vivo. Gene therapy...
by CheckRare Staff | Feb 1, 2023
Ying Huang, PhD, CEO of Legend Biotech, discusses updated results from the CARTITUDE-1 study and data from two cohorts in the CARTITUDE-2 study. Both of these studies are evaluating cilta-cel in patients with relapsed/refractory multiple myeloma. These data...
by CheckRare Staff | Jan 31, 2023
Conditioning Agent (JSP191) For Stem Cell Transplants Shows Promise in Patients With Fanconi Anemia Ronald Martell, President, and CEO of Jasper Therapeutics, discusses the positive data from the ongoing phase 1/2 study testing JSP191 as a conditioning...
