by Madaline Spencer | Feb 25, 2026
Joseph Muenzer, MD, PhD, Pediatric Biochemical Geneticist at University of North Carolina Chapel Hill, discusses results from a phase 1/2 study of tividenofusp alfa in patients with mucopolysaccharidosis II (MPS II; Hunter syndrome). MPS II is an...
by Madaline Spencer | Feb 19, 2026
Caroline Hastings, MD, Pediatric Hematology Oncology, Professor of Pediatrics at the University of California, San Francisco, discusses real-world outcomes of arimoclomol in patients with Niemann Pick type C (NPC). NPC is a complex lysosomal storage...
by Madaline Spencer | Feb 18, 2026
Ali Mohajer, PhD, Qral Group, discusses the age-stratified burden of post-allogeneic hematopoietic stem cell transplantation (post-allo-HSCT) complications in Hurler syndrome (MPS IH). MPS IH is the most severe form of mucopolysaccharidosis type I, caused by a...
by Madaline Spencer | Feb 17, 2026
Roberto Mina, MD, Associate Professor at Winship Cancer Institute of Emory University, discusses the Majestec-9 clinical trial of Tecvayli (teclistamab) in patients with multiple myeloma (MM). MM is a form of cancer due to abnormal and uncontrolled...
by Madaline Spencer | Feb 13, 2026
Tsewang Tashi, MD, Hematologist at the Huntsman Cancer Institute at the University of Utah, discusses long-term data from the PIONEER clinical trial examining the use of avapritinib in patients with indolent systemic mastocytosis (ISM). Systemic...
