FDA Approves Tazverik to Treat Rare Sarcoma

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...

Beta Thalassemia Major (Cooley’s Anemia)

  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...

Fanconi Anemia

  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...

FDA Approves Treatment for Thyroid Eye Disease

The U.S. Food and Drug Administration (FDA) has approved Tepezza (teprotumumab-trbw) for the treatment of adults with thyroid eye disease. This is the first drug approved for this rare condition. Thyroid eye disease is a common problem that develops in people with...