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.
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Dr Ozlem Goker-Alpan and Dr John Jefferies discuss the role of the cardiologist and best practices for managing Lysosomal Disorders
Incremental cost-effectiveness ratios were $126,000 and $281,000 per QALY for two simulation models from a societal perspective.
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Davide Martino, discusses the BASIS clinical trial, a phase 3 study testing marstacimab in patients with hemophilia without inhibitors.
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Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders.
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…
Kimberly Moran, PhD, MBA, Head of US Rare Diseases at UCB, discuses new treatment options for myasthenia gravis,
For hematologic malignancy (HM) patients admitted to the ICU, survival is 49 percent at seven days and 21 percent at 12 months.