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.
For hematologic malignancy (HM) patients admitted to the ICU, survival is 49 percent at seven days and 21 percent at 12 months.
Incremental cost-effectiveness ratios were $126,000 and $281,000 per QALY for two simulation models from a societal perspective.
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Treatment with NALL for 12 weeks yields better neurological status than placebo among patients with Niemann-Pick disease type C.
Cat Lutz, PhD, MBA, VP at JAX Rare Disease Translational Center, discusses rare disease research’s impact on understanding common diseases.
Bill Grambley explains how his company attacks health inequalities by providing personalized pharmaceutical care with an AI based platform.
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This symposium, led by Ozlem Goker-Alpan, MD of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) reviews the current state of newborn screening…
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.