by Madaline Spencer and Joe Haddad | Mar 17, 2026
Amel Karaa, MD, Genetics and General Metabolism, Director of the Mitochondrial Disease Program at Massachusetts General Hospital, discusses the PRIZM clinical trial of zagociguat in patients with MELAS. MELAS, or Mitochondrial Encephalomyopathy, Lactic...
by Madaline Spencer and Joe Haddad | Mar 13, 2026
Kristin McKay, President and Executive Director of Project Alive, discusses the real-world perspective on daily challenges of living with mucopolysaccharides II (MPS II). MPS II is an inherited disorder of carbohydrate metabolism that occurs almost exclusively...
by Madaline Spencer and Joe Haddad | Oct 30, 2025
The U.S. Food and Drug Administration (FDA) has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). The indication is for adult and pediatric patients one year of age and older with a susceptible nucleophosmin 1 (NPM1)...
by Madaline Spencer and Joe Haddad | Oct 14, 2025
May Lee Tjoa, PhD, Senior Global Medical Affairs Leader: Nipocalimab and Maternal-Fetal Immunology at Johnson & Johnson, discusses data on hemolytic disease of the fetus and newborn (HDFN) from the 2025 ISUOG World Congress on Ultrasound in Obstetrics and...
by Madaline Spencer and Joe Haddad | Oct 9, 2025
Jannine Williams, Compound Development Team Leader at Johnson & Johnson, discusses key takeaways from studies on hemolytic disease of the fetus and newborn (HDFN) presented at ISUOG World Congress 2025. HDFN is a rare condition characterized by the...
