by Madaline Spencer | Dec 22, 2025
Joseph Sullivan, MD, Professor of Neurology and Pediatrics at the University of California at San Francisco, discusses the results of the BUTTERFLY clinical trial in pediatric patients with Dravet syndrome. Dravet syndrome is the most severe of a group...
by Madaline Spencer | Dec 19, 2025
Shayla Bergmann, MD, Pediatric Hematologist/Oncologist at the Medical University of South Carolina, discusses phase 3 data of Vonvendi for the treatment of Von Willebrand disease (VWD). VWD is a genetic condition caused by a missing or defective clotting...
by Madaline Spencer | Dec 17, 2025
The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. gMG is a...
by Madaline Spencer | Dec 17, 2025
A recent study, published in the New England Journal of Medicine, looked at the effects of sotatercept for pulmonary arterial hypertension (PAH) within the first year after diagnosis. PAH affects the heart and lungs. It is characterized by abnormally high blood...
by Madaline Spencer | Dec 16, 2025
The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to less than 12 years of age. HAE is a condition characterized by...
