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The U.S. Food and Drug Administration approved L-glutamine oral powder (Endari, Emmaus Medical, Inc.) for oral administration to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years and older.
Approval was based on data from a randomized, double-blind, placebo-controlled, multi-center clinical trial (NCT01179217) enrolling 230 patients (5 to 58 years old) with sickle cell anemia or sickle β0-thalassemia who had two or more painful crises within the 12 months prior to enrollment. Eligible patients stabilized on hydroxyurea for at least 3 months, continued their therapy throughout the study. Patients were randomized to receive either L-glutamine or placebo for 48 weeks followed by three weeks of drug tapering.
education & researchDisability evaluation under social security (hematological disorders-adult)A. What hematological disorders do we ev...
education & researchHealth-related quality of life at 1 Year in the drepagreffe trial comparing chronic transfusion to transplantation i...Health is not just defined as the absenc...
videos & visualsSickle cell warrior documentaryhttps://www.youtube.com/watch?v=ZxC4vtN2...
news & eventsSCD Patients Benefit From Early Rivipansel Treatment for VOCs, New Analyses ShowStarting treatment with rivipansel (GMI-...
videos & visualsSickle Cell Disease and Marrow/Stem Cell Donationhttps://www.youtube.com/watch?v=mstThQc7...
news & eventsIronwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disea...Ironwood Pharmaceuticals, Inc., a commer...
videos & visualsDebbie’s Story on BBC2 Hospitalhttps://www.facebook.com/bbctwo/videos/1...
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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.