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...
by James Radke, PhD | Aug 30, 2022
Larry J Bauer, Senior Regulatory Drug Expert from Hyman, Phelps, & McNamara PC, a dedicated food and drug law firm, discusses the history and purposes of patient-focused drug development (PFDD) meetings. As Mr. Bauer explains, PFDD meetings are designed to...
by James Radke, PhD | Aug 29, 2022
Larry J Bauer, Senior Regulatory Drug Expert from Hyman, Phelps, & McNamara PC, a dedicated food and drug law firm, discusses an upcoming patient-focused drug development (PFDD) meeting on galactosemia. Galactosemia is a rare metabolism disorder that...
by James Radke, PhD | Aug 25, 2022
The U.S. Food and Drug Administration (FDA) has approved ibrutinib (Imbruvica) for pediatric patients ≥ 1 year of age with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy. cGVHD is a rare complication that may occur after...
