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NHS introduces Pfizer's tafamidis for ATTR-CM treatment

NHS introduces Pfizer's tafamidis for ATTR-CM treatment
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United Kingdom , Voxelotor Oxbryta , Susan Rienow , Simon Ray , Innovative Medicines Fund , National Institute Of Health , United Kingdom National Health Service , National Health Service , National Institute , Care Excellence , Pharmaceutical Technology ,

Sickle Cell Disease (Sickle Cell Anemia)

Sickle cell disease is the most common blood disorder passed down from parents to children. Learn how a gene mutation causes it. ....

United States , Voxelotor Oxbryta , What Is Sickle Cell , Cell Disease Causes , African American , Middle Eastern , Asian Indian , Sickle Cell ,

Pain Crises in Sickle Cell Disease: A Clinical Guide to Prevention and Treatment


Defining Sickle Cell Disease
Sickle cell disease (SCD) is the consequence of homozygosity for a single amino acid change in the beta-globin chain that results in structurally abnormal hemoglobin S, or by compound heterozygosity for hemoglobin S and another β-globin chain abnormality, typically hemoglobin C or beta-0 thalassemia. Hemoglobin SS (HbSS) and HbS beta-0 thalassemia present in a similar clinical manner and are commonly referred to together as sickle cell anemia (SCA), which is the most severe subtype of SCD.
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The sickling process causes secondary changes in cell shape, size, cation and water content, and membrane structure that contribute to the impairment of intrinsic cell deformability. ....

United States , Voxelotor Oxbryta , National Acute Chest Syndrome Study Group , World Health Organization , American Society Of Hematology , Centers For Medicare , Expert Panel , Medicaid Services , Blood Institute , American Society Of Hematologists , National Heart , Sota Omoigui , Sickle Cell Disease , Normalt Cell Expressed , Sickle Cell , Sickle Cell Crisis , West Africa , Sickle Cell Anemia , African American , Acute Pain Crisis , Chest Syndrome , Sickle Cell Pain , American Society , Sequal Eclipse , Without Walls , Acute Hospital Care At Home ,