by Peter Ciszewski | Oct 2, 2021
Pritesh J. Gandhi, PharmD, Former Vice President and General Manager, Lumasiran Program at Alnylam Pharmaceuticals, discusses how the severity of primary hyperoxaluria type 1 (PH1) impacts hospital visits. PH1 is a rare genetic disease in which excessive...
by Peter Ciszewski | Oct 1, 2021
Richard Colvin, MD, PhD, Vice President of Clinical Development and Interim Chief Medical Officer at Bluebird Bio, discusses the recent announcement by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) that they...
by Peter Ciszewski | Oct 1, 2021
The U.S. Food and Drug Administration (FDA) has approved maralixibat (Livmarli) for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. Alagille syndrome is an inherited disorder that can affect the liver, heart,...
by Peter Ciszewski | Oct 1, 2021
Julie Dohm, Of Counsel at Covington & Burling based in Silver Spring, Maryland, discusses some of the key differences between FDA-approved drugs versus compounded medications. Between 2016 and 2019, Julie served as the lead on compounding for the U.S. Food...
by Peter Ciszewski | Oct 1, 2021
Clete A. Kushida, MD, PhD, Neurologist and Division Chief and Medical Director of Stanford Sleep Medicine Center, discusses the signs and symptoms of narcolepsy. As Dr. Kushisa explains, narcolepsy is a rare neurological disorder characterized by excessive...
