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.
Alaa Hamed, MD, Global Head of Medical Affairs Rare Diseases at Sanofi, discusses the importance of rare disease registries.
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Robin Kate Kelley, professor of clinical medicine, University of California, San Francisco, dissects biliary tract cancer.
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Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases.
Project FIND-OUT enables the rapid detection of rare genetic neurodevelopmental disorders in babies ages 3-12 months.
Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett…
Monica Gadelha, DR, PhD, University of Rio De Janeiro, discusses her acromegaly research.
Davide Martino, discusses the BASIS clinical trial, a phase 3 study testing marstacimab in patients with hemophilia without inhibitors.
Monica Gadelha, DR, PhD, at the University of Rio De Janeiro discusses the PATHFNDR-1 trial and acromegaly.