by Peter Ciszewski | Jan 25, 2020
This week, numerous studies were published in peer-reviewed journals that highlight some of the innovative work that researchers around the world are doing to advance our understanding of rare conditions. Below are some of the highlights. Teprotumumab for the...
by Peter Ciszewski | Jan 24, 2020
The U.S. Food and Drug Administration (FDA) has approved Tazverik (tazemetostat) to treat persons 16 years and older with metastatic or locally advanced epithelioid sarcoma who are not eligible for complete resection. Epithelioid sarcoma is a rare form of soft tissue...
by Peter Ciszewski | Jan 24, 2020
Farzana Sayani, MD, Assistant Professor of Medicine at the Hospital of the University of Pennsylvania explains beta thalassemia major. Beta thalassemia major (aka Cooley’s anemia) is the most severe form of beta thalassemia. It is a genetic disorder involving...
by Peter Ciszewski | Jan 23, 2020
Gaurav Shah, MD, Chief Executive Officer and President of Rocket Pharmaceuticals, explains Fanconi anemia, a rare blood disorder that his company is developing a gene therapy to treat. Fanconi anemia is an inherited disorder involving mutations of one of many...
by Peter Ciszewski | Jan 22, 2020
Rod Humerickhouse, MD, PhD at Abbvie Pharmaceuticals highlights the positive long-term follow-up data showing patients with chronic lymphocytic leukemia (CLL) taking venetoclax in combination with rituximab (for relapsed CLL) or obinutuzumab (newly diagnosed...
