Sickle cell anemia

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Sickle cell anemia, a type of sickle cell disease (SCD), is a genetic condition that affects your red blood cells (RBCs). It affects around Healthy RBCs are donut-shaped. They’re flexible and can easily move through the tiniest of your blood vessels. However, if you have Sickle-shaped RBCs are prone to getting stuck in small vessels, making it hard for blood to reach many parts of your body. This can cause pain, infections, and tissue damage. Until recently, Because of these factors, current treatment options are often not possible or recommended for people with SCD. However, a new cure for SCD has recently appeared on the horizon: gene therapy. What is it, and when can you receive this treatment? Read on to find out. Each of your cells contains These instructions can sometimes have typos, or mutations. Most of the time, mutations aren’t of major consequence, but sometimes they can hit critical parts of your genes. This can harm your cells’ ability to properly perform their assigned task. This is what happens in SCD. Gene therapy uses specialized molecular tools called There are a couple of ways Gene editing In this approach, scientists use CRISPR-Cas9 to cut your DNA at the sites of the mutations and replace them with the “correct” code. This is called gene editing based on its similarity to the work of a book editor. When used in SCD, CRISPR-Cas9 edits mutations in your hemoglobin genes. This restores hemoglobin’s ability to capture oxygen and gives RBCs back their healt...

Overview Sickle cell anemia is one of a group of inherited disorders known as sickle cell disease. It affects the shape of red blood cells, which carry oxygen to all parts of the body. Red blood cells are usually round and flexible, so they move easily through blood vessels. In sickle cell anemia, some red blood cells are shaped like sickles or crescent moons. These sickle cells also become rigid and sticky, which can slow or block blood flow. There's no cure for most people with sickle cell anemia. Treatments can relieve pain and help prevent complications associated with the disease. Symptoms Signs and symptoms of sickle cell anemia usually appear around 6 months of age. They vary from person to person and may change over time. Signs and symptoms can include: • Anemia. Sickle cells break apart easily and die. Red blood cells usually live for about 120 days before they need to be replaced. But sickle cells typically die in 10 to 20 days, leaving a shortage of red blood cells (anemia). Without enough red blood cells, the body can't get enough oxygen and this causes fatigue. • Episodes of pain. Periodic episodes of extreme pain, called pain crises, are a major symptom of sickle cell anemia. Pain develops when sickle-shaped red blood cells block blood flow through tiny blood vessels to your chest, abdomen and joints. The pain varies in intensity and can last for a few hours to a few days. Some people have only a few pain crises a year. Others have a dozen or more a year. A s...

Family physicians are the primary and sometimes only health care resource for families affected by sickle cell disease. Recently published guidelines provide important recommendations for health maintenance, acute care, and monitoring of disease-modifying therapy in persons with this condition. This overview highlights some of the most important clinical activities that can and should be carried out in the community care setting. Children with sickle cell anemia should receive prophylactic penicillin from birth through at least five years of age, and all persons with sickle cell disease require vaccination to prevent invasive pneumococcal disease. Annual screening with transcranial Doppler ultrasonography is recommended for all children with sickle cell disease beginning at two years of age and continuing through adolescence to evaluate the risk of stroke and to initiate transfusion therapy in those at high risk. Vasoocclusive crises require immediate and adequate analgesia appropriate to the level of patient-reported pain. Antibiotics, hospitalization, and incentive spirometry are indicated for those with acute chest syndrome. There is strong evidence to support the promotion and use of hydroxyurea therapy in patients nine months and older who have sickle cell anemia because its use can decrease the frequency of vasoocclusive crises and acute chest syndrome with limited adverse effects. Family physicians and family medical homes are essential to the care of children, adul...

Overview Anemia is a problem of not having enough healthy red blood cells or hemoglobin to carry oxygen to the body's tissues. Hemoglobin is a protein found in red cells that carries oxygen from the lungs to all other organs in the body. Having anemia can cause tiredness, weakness and shortness of breath. There are many forms of anemia. Each has its own cause. Anemia can be short term or long term. It can range from mild to severe. Anemia can be a warning sign of serious illness. Treatments for anemia might involve taking supplements or having medical procedures. Eating a healthy diet might prevent some forms of anemia. • • • • • Symptoms Anemia symptoms depend on the cause and how bad the anemia is. Anemia can be so mild that it causes no symptoms at first. But symptoms usually then occur and get worse as the anemia gets worse. If another disease causes the anemia, the disease can mask the anemia symptoms. Then a test for another condition might find the anemia. Certain types of anemia have symptoms that point to the cause. Possible symptoms of anemia include: • Tiredness. • Weakness. • Shortness of breath. • Pale or yellowish skin, which might be more obvious on white skin than on Black or brown skin. • Irregular heartbeat. • Dizziness or lightheadedness. • Chest pain. • Cold hands and feet. • Headaches. When to see a doctor Make an appointment with your health care provider if you're tired or short of breath and don't know why. Low levels of the protein in red blood cel...

What is sickle cell anemia? Sickle cell anemia, or sickle cell disease (SCD), is a genetic disease of the red blood cells (RBCs). Normally, RBCs are shaped like discs, which gives them the flexibility to travel through even the smallest blood vessels. However, with this disease, the RBCs have an abnormal crescent shape resembling a sickle. This makes them sticky and rigid and prone to getting trapped in small vessels, which blocks blood from reaching different parts of the body. This can cause pain and tissue damage. SCD is an autosomal recessive condition. You need two copies of the gene to have the disease. If you have only one copy of the gene, you are said to have sickle cell trait. Symptoms of sickle cell anemia usually show up at a young age. They may appear in babies as early as 4 months old, but generally occur around the 6-month mark. While there are multiple types of SCD, they all have similar symptoms, which vary in severity. These include: • excessive fatigue or irritability, from anemia • fussiness, in babies • bedwetting, from associated kidney problems • jaundice, which is yellowing of the eyes and skin • swelling and pain in hands and feet • frequent infections • pain in the chest, back, arms, or legs Hemoglobin is the protein in red blood cells that carries oxygen. It normally has two alpha chains and two beta chains. The four main types of sickle cell anemia are caused by different mutations in these genes. Hemoglobin SS disease Hemoglobin SS disease is t...