by Madaline Spencer and Scott Harwood | Mar 6, 2026
John Taggart, Head of Communications at Niemann-Pick UK (NPUK), discusses the ASMD Perspective Index and its use in diagnosing Niemann-Pick disease. Niemann-Pick disease is an inherited condition characterized by abnormal lipid metabolism that causes harmful...
by Madaline Spencer and Scott Harwood | Mar 5, 2026
Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). Mr. Stevens has been involved with the UK MPS...
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...
