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Approved by the US Food and Drug Administration (FDA) in July 2017 to reduce the acute vaso-occlusive pain complications of sickle cell disease, L-glutamine oral powder (Endari) was the first drug approved for the rare blood disease in the 20 years since the approval of hydroxyurea.
Now, the New England Journal of Medicine (NEJM) has published the 48-week phase 3 clinical trial results that supported the FDA approval. From the data acquired in the phase 3 trial, investigators concluded that the median number of pain crises in children and adults with sickle cell disease was lower among those who received oral therapy with l-glutamine, administered alone, or with hydroxyurea, compared with those who received placebo, with or without hydroxyurea.
“The idea that this [publication] comes a year after [approval] really reflects the vigor of the review process and shows NEJM really did its job of assuring the public interest that L-glutamine, along with the findings of the study, are real and significant,” Darrell W. Harrington, MD, MACP, chief medical officer at Emmaus Life Sciences, told Rare Disease Report®.
news & eventsEmmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for ...Emmaus Life Sciences, Inc., anno...
news & eventsEmmaus Life Sciences, Inc. to present results of phase 3 study of endariTM (L-glutamine oral powder) at 59th America...Emmaus will present results of a Phase 3...
news & eventsEmmaus Life Sciences Receives Validation From European Medicines Agency on Marketing Authorization Application for S...Emmaus Life Sciences, Inc. announced tod...
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
education & researchPhase 3 study of L-Glutamine in sickle cell disease: Analyses of time to first and second crisis and average cumulat...A Phase 3, double-blind, randomized, pla...
education & researchPhase 3 Study of L-Glutamine Therapy in Sickle Cell Anemia and Sickle ß0-Thalassemia Subgroup Analyses Show Consist...Background: Sickle cell disease (SCD) is...
news & eventsEndari reduces pain crises, hospitalizations in sickle cell patients, phase 3 trial showsTreatment with Endari (L-glutamine) le...
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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.