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...
by Madaline Spencer | Aug 1, 2025
Henry Wong, MD, PhD, Dermatologist at the San Diego Veterans Hospital and the University of California San Diego (UCSD), discusses cutaneous T-cell lymphoma (CTCL). CTCL is a rare group of malignancies that attack the body’s immune lymphatic system, affecting...
