by Peter Ciszewski | Oct 7, 2021
Matthew Klein, MD, MS, FACS, Chief Development Officer at PTC Therapeutics, provides an overview of phenylketonuria (PKU). As Dr. Klein explains, PKU is a rare genetic metabolic disorder caused by a defect in the gene involved in the production of phenylalanine...
by Peter Ciszewski | Oct 6, 2021
Salvatore Dileo, RPh, Director of Pharmacy, Westmed Medical Group in New York, discusses the differences between compounded medications versus FDA-approved drugs. Compounded drugs can serve an important medical need for patients, but they do not have the same...
by Peter Ciszewski | Oct 6, 2021
Samantha Masterson, CEO and President of Myasthenia Gravis Foundation of America (MGFA), gives advice to newly diagnosed myasthenia gravis patients. Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal...
by Peter Ciszewski | Oct 5, 2021
Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the latest results from the COMET study assessing the efficacy of a new enzyme replacement therapy (ERT) to treat late-onset Pompe disease. Pompe disease is a...
by Peter Ciszewski | Oct 5, 2021
Chelsea Johnson, mother of a boy with hypothalamic obesity (HO), discusses the lack of understanding about this disease. HO is a rare endocrine disorder characterized by severe and debilitating obesity. Signs and symptoms of hypothalamic obesity include...
