by Madaline Spencer | Nov 25, 2025
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib was first approved in 2020 for pediatric...
by Madaline Spencer | Nov 24, 2025
The U.S. Food and Drug Administration (FDA) has updated the prescribing information for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). DMD is a rare genetic, neuromuscular condition characterized by progressive muscle wasting. Symptoms...
by Madaline Spencer | Nov 21, 2025
The U.S. Food and Drug Administration (FDA) has approved Redemplo (plozasiran) for the treatment of adult patients with familial chylomicronemia syndrome (FCS). FCS is an inherited condition that disrupts the normal breakdown of fats in the body, causing large amounts...
by Madaline Spencer | Nov 20, 2025
The U.S. Food and Drug Administration (FDA) has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation. Acute myeloid leukemia (AML) is a cancer that affects the blood and bone...
by Madaline Spencer | Nov 18, 2025
Aman Chauhan, MD, Oncologist at the University of Miami, discusses details of a phase 3 study testing paltusotine in patients with carcinoid syndrome due to neuroendocrine tumors (NETs). NETs are rare tumors that usually develop in the GI tract or lungs,...
