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...
by James Radke, PhD | Aug 24, 2022
Jennifer McNary, Head of Patient Advocacy and Engagement at Fulcrum Therapeutics, and mother to two boys with Duchenne muscular dystrophy, discusses the importance of clinically meaningful primary outcomes in clinical trials. Fulcrum Therapeutics is enrolling...
by James Radke, PhD | Aug 15, 2022
Guillaume Canaud, MD, PhD, of the Paris Descartes University, explains why it is important to regularly measure objective outcomes in persons with PiK3CA-related overgrowth syndrome (PROS). PROS is a group of rare congenital disorders that lead to the...
