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...

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...

Award Winning Castleman Disease Study

  Ruth-Anne Langan Pai, collaborative research project lead for the Castleman Disease Collaborative Network (CDCN) , and a  PhD student at the University of Pennsylvania explains her award-winning study that was presented at the American Society of Hematology...

Facioscapulohumeral Muscular Dystrophy

  Diego Cadavid, MD of Fulcrum Therapeutics explains facioscapulohumeral muscular dystrophy, a rare disabling disease characterized by progressive skeletal muscle loss. The disease generally begins with weakness in facial muscles but as the condition progresses, ...

Future Treatment Options for Multiple Myeloma

  Amrita Krishnan, MD, hematologist-oncologist at the City of Hope Comprehensive Cancer Center, talks about what the future holds for managing people with multiple myeloma. Multiple myeloma is a rare hematologic cancer characterized by the expansion of malignant...
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