FDA Fast Tracks Approval of Voxelotor to Treat Underlying Cause of Sickle Cell Disease | CheckRare.com
The U.S. Food and Drug Administration (FDA) has approved Oxbryta (voxelotor) to treat persons, 12 years and older, with sickle cell disease.
The U.S. Food and Drug Administration (FDA) has approved Oxbryta (voxelotor) to treat persons, 12 years and older, with sickle cell disease.
Alpha-mannosidosis is a rare genetic disorder characterized by a deficiency of the enzyme alpha-D-mannosidase.
Richard F. Riedel, MD, Medical Oncologist, Duke Health, discusses tyenosynovial giant cell tumors (TGCT)
Robin Kate Kelley, MD, Professor of Clinical Medicine Hematology/IOncology, University of California, San Francisco, describes the need for better treatment plans for patients with biliary…
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
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Approval allows Onivyde (plus oxaliplatin, fluorouracil, and leucovorin) as first-line therapy.