by CheckRare Staff | Feb 20, 2023
Henry Kaminski, MD, Professor of Medicine at The George Washington University ,provides a brief history lesson on our understanding of myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease. Common symptoms include weakness of the muscles that...
by CheckRare Staff | Feb 16, 2023
Sangamo Therapeutics’ Focus on Rare Diseases Nathalie Dubois-Stringfellow, PhD, Senior Vice President of Product Development & Management at Sangamo Therapeutics discusses the company’s focus on rare diseases. Sangamo Therapeutics, Inc has several...
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...
