Novartis gets FDA ok for sickle-cell drug

Novartis announced it won U.S. FDA approval for its experimental sickle cell disease drug, Adakveo.
Source: - Category: Pharmaceuticals Source Type: news

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An 18-year-old white woman was referred to our service with complaint of “ulcerations on the tongue.” The lesions worsened in the last 6 months when the orthodontic appliance was placed. The patient was vegetarian, and she reported a prolonged menstrual period. Oral examination revealed multiple symptomatic ulcerations with irregular shape with pseudomembrane involvi ng the lower lip, tongue, and buccal mucosa bilaterally. Laboratory tests were requested, which showed sickle cell trait of anemia with presence of microcytic red blood cells as well as iron deficiency and ferritin.
Source: Oral Surgery, Oral Medicine, Oral Pathology, Oral Radiology, and Endodontics - Category: ENT & OMF Authors: Source Type: research
Authors: Croisé F, Le Lez ML, Pisella PJ Abstract INTRODUCTION: Sickle cell retinopathy is the main ophthalmologic complication of sickle cell syndrome. Optical coherence tomography (OCT) and optical coherence tomography-angiography (OCT-A) permit demonstration of central retinal involvement. The goal of this study is to determine whether central retinal involvement is predictive of peripheral retinal ischemia. MATERIALS AND METHODS: We carried out a retrospective study of 31 patients with sickle cell disease who underwent a complete ophthalmologic examination. We focused on capillary density of the supe...
Source: Journal Francais d Ophtalmologie - Category: Opthalmology Tags: J Fr Ophtalmol Source Type: research
Abstract Hematopoietic stem cell transplantation is a life-saving therapy for many patients with cancer, as well as patients with some nonmalignant hematologic disorders, such as aplastic anemia, sickle cell disease, and certain congenital immune deficiencies. Kidney injury directly associated with stem cell transplantation includes a wide range of structural and functional abnormalities, which may be vascular (hypertension, thrombotic microangiopathy), glomerular (albuminuria, nephrotic glomerulopathies), and/or tubulointerstitial. AKI occurs commonly after stem cell transplant, affecting 10%-73% of patients. The...
Source: Clinical Journal of the American Society of Nephrology : CJASN - Category: Urology & Nephrology Authors: Tags: Clin J Am Soc Nephrol Source Type: research
Due to the high number of transfusions which patients with hereditary hemoglobinopathies (thalassemia, sickle cell disease) receive, they represent high risk of acquiring parenterally transmitted infectious diseases. In this respect, non pathogenic human commensal viruses, which also demonstrate parenteral transmission routes might also be acquired.One of the most widely spread parenterally-transmitted human commensal viruses include the Human Pegivirus-1 (HPgV-1, GBV-C) and Torque teno viruses (TTV) including its SEN virus-like (SENV) forms.
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
Familial haploidentical stem-cell transplant improves outcomes in children and adolescents with high-risk sickle-cell disease, researchers report.Reuters Health Information
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Medscape Today News Source Type: news
rodel Sickle cell disease (SCD) is a group of inherited disorders affecting red blood cells, which is caused by a single mutation that results in substitution of the amino acid valine for glutamic acid in the sixth position of the β-globin chain of hemoglobin. These mutant hemoglobin molecules, called hemoglobin S, can polymerize upon deoxygenation, causing erythrocytes to adopt a sickled form and to suffer hemolysis and vaso-occlusion. Until recently, only two drug therapies for SCD, which do not even fully address the manifestations of SCD, were approved by the United States (US) Food and Drug Administration...
Source: Molecules - Category: Chemistry Authors: Tags: Review Source Type: research
Oral arginine supplementation significantly increased plasma arginine levels and improved acute pain-related outcomes in children with sickle cell disease in a randomized, placebo-controlled, phase 2 trial.Medscape Medical News
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Pediatrics News Source Type: news
Abstract Ischemia-reperfusion (I/R) injury is a serious condition which is associated with myocardial infarction, stroke, acute kidney injury, trauma, circulatory arrest, sickle cell disease, and sleep apnea and can lead to high morbidity and mortality. Salts of zinc (Zn) are commonly used by humans and have protective effects against gastric, renal, hepatic, muscle, myocardial, or neuronal ischemic injury. The present review evaluates molecular mechanisms underlying the protective effects of Zn supplement against I/R injury. Data of this review have been collected from the scientific articles published in databas...
Source: Biological Trace Element Research - Category: Biology Authors: Tags: Biol Trace Elem Res Source Type: research
Scientists report progress using gene therapy to treat sickle cell disease, a common and devastating genetic blood disorder.
Source: NPR Health and Science - Category: Consumer Health News Authors: Source Type: news
A point-of-care (POC) test for sickle cell disease was highly accurate and improved newborn screening when combined with a laboratory diagnostic, according to a recent Pediatricsstudy involving 2159 infants in Haiti.
Source: JAMA - Category: General Medicine Source Type: research
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