by Madaline Spencer and Scott Harwood | Mar 4, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the importance of newborn screening and the need for more effective treatments in infantile Krabbe disease. Krabbe disease is a lysosomal disorder that affects the entire body,...
by Scott Harwood | Nov 11, 2025
The U.S. Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). HR-SMM and Darzalex Faspro HR-SMM is an asymptomatic blood and bone...
by Madaline Spencer and Scott Harwood | Oct 27, 2025
The U.S. Food and Drug Administration (FDA) has approved Blenrep (belantamab mafodotin) in combination with bortezomib and dexamethasone (BVd) for the treatment of adult patients with relapsed/refractory multiple myeloma (MM). The indication is for those who have...
by Madaline Spencer and Scott Harwood | Oct 13, 2025
Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP). ITP is a bleeding disorder characterized by low levels of platelets. It is an autoimmune disorder in...
by Scott Harwood | Mar 4, 2025
Heather Lau, MD, Executive Director of Global Clinical Development at Ultragenyx, discusses positive results regarding gene therapy UX111 for treatment of patients with Sanfilippo syndrome type A (MPS IIIA). MPS IIIA is a severe, progressive disorder that...
