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...
by Madaline Spencer | Aug 4, 2025
The U.S. Food and Drug Administration (FDA) has approved Empaveli (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients ages 12 years and older to reduce proteinuria. C3G...
by Madaline Spencer | Aug 1, 2025
The U.S. Food and Drug Administration (FDA) has approved Skytrofa (lonapegsomatropin-tcgd; TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). It was approved for the treatment of pediatric GHD by the FDA in...
