by Madaline Spencer | Sep 30, 2026
The US Food and Drug Administration (FDA) has approved Emcitate (tiratricol) for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency. MCT8 deficiency is a rare disorder of brain development that causes moderate...
by Madaline Spencer and Ailene Cantelmi | Sep 30, 2026
A study published in The Lancet evaluated the safety and efficacy of ralinepag in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the heart and lungs, characterized by abnormally high blood pressure (hypertension) in the...
by Madaline Spencer | Sep 30, 2026
The US Food and Drug Administration (FDA) has approved Atebrioz (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification (HO) in patients ages 12 years and older with fibrodysplasia ossificans progressiva (FOP). FOP is a disorder in which...
by Madaline Spencer | Sep 29, 2026
The US Food and Drug Administration (FDA) has approved Lyrfigtu (lirafugratinib) for the treatment of cholangiocarcinoma with FGFR2 fusion or other rearrangement. Cholangiocarcinoma is a rare, aggressive cancer that forms in the bile duct. Patients with...
by Madaline Spencer | Sep 28, 2026
Amy Waldman, MD, pediatric neurologist and Medical Director of the Leukodystrophy Center at the Children’s Hospital of Philadelphia, discusses the approval of Zanvastro (zilganersen) to treat patients with Alexander disease. Alexander disease is a rare...
