by Madaline Spencer | Jun 22, 2026
Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses results from a phase 3 study of Voxzogo (vosoritide) in children with hypochondroplasia. Hypochondroplasia is a rare skeletal disease characterized by very short...
by Madaline Spencer | Jun 19, 2026
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses results from the phase 2 ElevAATe clinical trial of efdoralprin alfa in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited disease that causes an...
by Madaline Spencer | Jun 18, 2026
Yi Lin, MD, PhD, Hematologist/Oncologist at Mayo Clinic, discusses immune effector cell-associated enterocolitis (IEC-EC) in patients with relapsed/refractory multiple myeloma (R/R MM) treated with ciltacabtagene autoleucel (cilta-cel). MM is a rare...
by Madaline Spencer | Jun 16, 2026
The US Food and Drug Administration (FDA) has approved an expanded indication for Hympavzi (marstacimab) to include treatment of patients with hemophilia A or B who are 12 years and older with inhibitors, as well as pediatric patients (ages 6 to 11 years) with or...
by Madaline Spencer | Jun 16, 2026
Carolina Barnett-Tapia, MD, Neuromuscular Neurologist and the University of Toronto, discusses the safety and efficacy of subcutaneous efgartigimod PH20 in ocular myasthenia gravis (oMG). oMG is a neuromuscular disease characterized by autoantibody...
