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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Ozlem Goker-Alpan, MD, Founder and CMO of LDRTC, discusses the importance of newborn screening for lysosomal disorders.
Judith Luker discusses the sudden unexpected death in epilepsy communication gap and the importance of physician-patient conversations.
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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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