by Madaline Spencer | Aug 8, 2025
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab) injection as a once-daily prophylaxis treatment to prevent or reduce frequency of bleeding episodes in patients ages 12 years and older with hemophilia A or B without inhibitors. This is an...
by Madaline Spencer | Aug 7, 2025
Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses the organization’s goals and upcoming clinical trials. The ARISE...
by Madaline Spencer | Aug 6, 2025
James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses age in risk stratification in patients with mutant isocitrate dehydrogenase (mIDH) glioma. mIDH is a rare metabolic gene that causes tumor initiation in many gliomas and...
by Madaline Spencer | Aug 5, 2025
The U.S. Food and Drug Administration (FDA) has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that increases the body’s levels of phenylalanine. Humans cannot make phenyalanine, but it is a...
by Madaline Spencer | Aug 4, 2025
Henry J. Kaminski, MD, Professor of Neurology at The George Washington University, Lead of the Myasthenia Gravis Rare Disease Network (MGNET), explains “Myasthenia Gravis: The Future Is Here,” an article discussing advancements in the treatment of myasthenia gravis...
