by James Radke, PhD | Sep 30, 2022
The US Food and Drug Administration (FDA) has approved the combination oral medication of sodium phenylbutyrate and taurursodiol (Relyvrio) to treat individuals with amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease, characterized by the...
by James Radke, PhD | Sep 29, 2022
The U.S. Food and Drug Administration (FDA) has approved dupilumab to treat adults with prurigo nodularis. Prurigo nodularis is an inflammatory skin disorder characterized by extreme itching and symmetrical rashes. Most areas commonly affected are the arms, legs, the...
by James Radke, PhD | Sep 19, 2022
The U.S. Food and Drug Administration (FDA) has granted accelerated approval for elivaldogene autotemcel (el-cel) to treat males 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). CALD is a progressive neurodegenerative disease that involves...
by James Radke, PhD | Sep 16, 2022
David Trainor, parent of an adult son with galactosemia, discusses the recent publication of Navigating Galactosemia Life Stages: A Handbook for the Galactosemia Community. Galactosemia is a rare metabolism disorder that affects the body’s ability to convert...
by James Radke, PhD | Sep 3, 2022
The U.S. Food and Drug Administration (FDA) has approved olipudase alfa for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD). ASMD is an autosomal recessive genetic disorder caused by mutations in the SMPD1 gene. That...
