by Peter Ciszewski | Oct 12, 2021
Dawn Laney, MS, CGC, CCRC, Assistant Professor, and Director of Emory Genetic Clinical Trials Center, discusses the benefits and drawbacks of screening newborns for Fabry disease. Fabry disease is a rare X-linked lysosomal storage disorder that results in the...
by Peter Ciszewski | Oct 12, 2021
Daniel Lovell, MD, MPH, Associate Director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center, provides an overview of juvenile idiopathic arthritis (JIA). As Dr. Lovell explains, JIA is an umbrella term for a number of...
by Peter Ciszewski | Oct 12, 2021
Julie Dohm discusses some of the adverse events that have associated with compounded drugs. Julie Dohm focuses on regulatory matters for pharmaceutical and biotechnology companies. Between 2016 and 2019, Julie served as the lead on compounding for the U.S. Food and...
by Peter Ciszewski | Oct 11, 2021
Clete A. Kushida, MD, PhD, Neurologist and Division Chief and Medical Director of Stanford Sleep Medicine Center, discusses the REST-ON clinical trial of FT218 for the treatment of narcolepsy. Narcolepsy is a rare neurological disorder characterized by excessive...
by Peter Ciszewski | Oct 10, 2021
Deborah Marsden, MD, Global Medical Expert, Medical Affairs, Ultragenyx, discusses the diagnostic journey for many patients with mucopolysaccharidosis type VII (MPS VII). MPS VII is a rare lysosomal storage disorder. The severity of MPS VII varies widely among...
