by Peter Ciszewski | Sep 1, 2021
Mary Dunkle, Senior Advisor for the National Organization for Rare Disorders (NORD), discusses the upcoming NORD Breakthrough Summit 2021 being held October 18-19, 2021. As Ms. Dunkle explains, the summit is one of the most important conferences in rare...
by Peter Ciszewski | Aug 31, 2021
Avanish Vellanki, Cofounder and CEO at Rain Therapeutics, describes the mechanism of action of milademetan (RAIN-32), an investigational MDM2 inhibitor which is currently being studied as a treatment for dedifferentiated liposarcoma in a phase 3 clinical trial....
by Peter Ciszewski | Aug 30, 2021
Sima Rozati, MD, Assistant Professor of Dermatology at the Johns Hopkins University School of Medicine, discusses how cutaneous T-cell lymphoma (CTCL) affects African American subpopulations. CTCL belongs to the non-Hodgkin lymphoma family as a rare group of...
by Peter Ciszewski | Aug 27, 2021
Michael J. Thorpy, MD, ChB, Director of the Sleep-Wake Disorders Center at the Montefiore Medical Center, discusses how narcolepsy is typically diagnosed. As Dr. Thorpy explains, narcolepsy is a rare neurological disorder characterized by excessive sleepiness...
by Peter Ciszewski | Aug 26, 2021
Omar Sinno, MD, Medical Lead of Rare Disease at UCB, gives an overview of myasthenia gravis. As Dr. Sinno explains, myasthenia gravis is a chronic autoimmune neuromuscular disease due to antibodies attacking a person’s neuromuscular template. It is...
