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Top Sickle Cell Disease News News

Maryland · MarylandThe U.S. Food and Drug Administration has granted orphan drug status to Sanofi’s rilzabrutinib, an investigational therapy aimed at preventing painful vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). This designation intends to encourage companies to develop therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides several incentives, such as exemption from certain fees, tax credits f…Read Article
FDA grants orphan drug status to oral inhibitor rilzabrutinib for SCD

Hospitals · MarylandAn algorithm that grew out of a collaboration between different specialties at the Medical University of South Carolina (MUSC) may help reduce unnecessary hospital admissions for children with sickle cell disease (SCD), according to a new study. “This algorithm safely allows our care of these patients to align with national thoughts and practice, while also considering quality of life for our patients and their families,” Shayla Bergmann, MD, pe…Read Article