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...
by Scott Harwood | Mar 3, 2025
Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with generalized myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles....
by Madaline Spencer | Feb 27, 2025
Alix Arnaud, Director of Health Economic and Value Assessment BP at Sanofi, discusses the effects of caplacizumab combination therapy in patients with acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare blood disorder characterized by low...
by Madaline Spencer | Feb 26, 2025
Aneal Khan, MD, President of MAGIC Clinic (Metabolics and Genetics in Canada), discusses five year end-of-study results from the FACTs trial in patients in Fabry disease. Fabry disease is a rare lysosomal storage disease characterized by a deficiency of...
by Madaline Spencer | Feb 25, 2025
David Meeker, MD, Chief Executive Officer of Rhythm Pharmaceuticals, discusses the U.S. Food and Drug Administration’s (FDA) approval of setmelamotide for children 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically...
