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Fast-track research focused on developing a new gene-modifying sickle cell disease treatment at the Medical University of South Carolina could lead to human clinical trials in as few as three years.
Patrick Woster, the SmartState endowed chair in medicinal chemistry at MUSC, said the project takes aim at the enzyme lysine-specific demethylase 1, or LSD 1. That enzyme is part of the biological process that makes mutated hemoglobin, leading to sickle-shaped blood cells. They stick together, causing extremely painful and dangerous blood flow problems. Inhibiting LSD 1, making it less effective, could turn an important gene back on again and keep sickle-shaped cells from sticking together.
education & researchMedical Marijuana Certification for Patients With Sickle Cell Disease: A Report of a Single Center ExperienceMore than one-third of adults with sickl...
education & researchThe influence of the age of adults with sickle cell disease on the uptake, utilization and efficacy of HydroxyureaDespite the efficacy of Hydroxyurea (HU)...
news & eventsMusic therapy helps relieve pain in adults with sickle cell disease, pilot trial suggestsMusic therapy can help ease pain in ad...
videos & visualsA Child Prodigy, a Painful Disease, and a Life-Changing Treatmenthttps://www.youtube.com/watch?v=SpzjD1ct...
news & eventsSiklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
news & eventsEmmaus, a leader in sickle cell disease treatment, signs agreement with Cardinal Health to solidify distribution net...Emmaus Life Sciences, Inc. announces...
news & eventsFDA Awards $2M to Phase 2 Trial of Vitamin D for Reducing Risk of SCD Respiratory ComplicationsThe U.S. Food and Drug Administration (F...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.