A New Era for Sickle Cell Disease, Acute Porphyria, and More

By Basem M. William, MD On Monday, the Director of the Division of Hematology Products in the Office of Hematology and Oncology Products at the U.S. Food and Drug Administration (FDA), Ann Farrell, MD, chaired the two special ASH-FDA Joint Symposia focusing on new...
Erythroferrone: A Bone to Pick with Thalassemia

Erythroferrone: A Bone to Pick with Thalassemia

By Alexander Glaros, MD, and Ahmar U. Zaidi, MD If the results of the past five years are any indication, the era of gene therapy is truly at hand, and nearly 100 years after Thomas Cooley first described the skeletal and morphological changes of the disease in a pair...

Oh, How Far We’ve Come!

By Janice Staber, MD Since I was little, I’ve been surrounded by scientific intrigue. One of my first memories of the idea of moving genetic material into a person was from my early high school days. When discussing gene transfer, a classmate asked, “So, we could make...