by Madaline Spencer | Nov 5, 2025
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the ElevAATe clinical trial of efdoralprin alfa for the treatment of patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited disease that...
by Madaline Spencer | Nov 4, 2025
Xiaohui Niu, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, discusses a long-term analysis of the MANEUVER clinical trial of pimicotinib for the treatment of patients with tenosynovial giant cell tumor (TGCT)....
by Madaline Spencer | Nov 3, 2025
The U.S. Food and Drug Administration (FDA) has approved an update to the product label of Winrevair (sotatercept) for the treatment of adults with pulmonary arterial hypertension (PAH). PAH is a rare lung and heart condition characterized by abnormally high blood...
by Madaline Spencer | Oct 31, 2025
Philip J. Ferrone, MD, Vitreoretinal Consultants of New York, discusses results from the TEASE-2 clinical trial of gildeuretinol in patients with Stargardt disease. Stargardt disease is a genetic eye disorder that causes progressive vision loss. It...
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)...
