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Elivaldogene Autotemcel
Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird bio and was given breakthrough therapy designation by the U.S. Food and Drug Administration in May 2018. Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells. It was approved for medical use by the U.S. Food and Drug Administration in September 2022. Medical uses Elivaldogene autotemcel is indicated for the treatment of people with early, active CALD in boys aged 4 to 17 for whom a matched hematopoietic stem cell donor is not available. Early, active CALD refers to asymptomatic or mildly symptomatic boys with gadolinium enhancement on brain Magnetic resonance imaging, MRI and a Loes score of 0.5-9, a scale that rates the severity of CALD white matter lesions on a scale of 0 (normal) to 34 (abnormal) in adrenoleukodystrophy. Elivaldogene autotemcel is a form ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravenous Infusion
Intravenous therapy (abbreviated as IV therapy) is a medical technique that administers fluids, medications and nutrients directly into a person's vein. The intravenous route of administration is commonly used for rehydration or to provide nutrients for those who cannot, or will not—due to reduced mental states or otherwise—consume food or water per os, by mouth. It may also be used to administer pharmaceutical drug, medications or other medical therapy such as blood transfusion, blood products or electrolytes to correct electrolyte imbalances. Attempts at providing intravenous therapy have been recorded as early as the 1400s, but the practice did not become widespread until the 1900s after the development of techniques for safe, effective use. The intravenous route is the fastest way to deliver medications and fluid replacement throughout the body as they are introduced directly into the circulatory system and thus quickly distributed. For this reason, the intravenous route o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Macrophage
Macrophages (abbreviated as M φ, MΦ or MP) ( el, large eaters, from Greek ''μακρός'' (') = large, ''φαγεῖν'' (') = to eat) are a type of white blood cell of the immune system that engulfs and digests pathogens, such as cancer cells, microbes, cellular debris, and foreign substances, which do not have proteins that are specific to healthy body cells on their surface. The process is called phagocytosis, which acts to defend the host against infection and injury. These large phagocytes are found in essentially all tissues, where they patrol for potential pathogens by amoeboid movement. They take various forms (with various names) throughout the body (e.g., histiocytes, Kupffer cells, alveolar macrophages, microglia, and others), but all are part of the mononuclear phagocyte system. Besides phagocytosis, they play a critical role in nonspecific defense (innate immunity) and also help initiate specific defense mechanisms (adaptive immunity) by recruiting other immune ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Anorexia (symptom)
Anorexia is a medical term for a loss of appetite. While the term in non-scientific publications is often used interchangeably with anorexia nervosa, many possible causes exist for a loss of appetite, some of which may be harmless, while others indicate a serious clinical condition or pose a significant risk. Anorexia is a symptom, not a diagnosis. Anorexia is not to be confused with the mental health disorder anorexia nervosa. Because the term 'anorexia' is often used as a short-form of anorexia nervosa, to avoid confusion a provider must clarify to a patient whether they are simply referring to a decreased appetite or the mental health disorder. Anyone can manifest anorexia as a loss of appetite, regardless of their gender, age, or weight. The symptom also occurs in other animals, such as cats, dogs, cattle, goats, and sheep. In these species, anorexia may be referred to as inappetence. As in humans, loss of appetite can be due to a range of diseases and conditions, as well as ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Dimethyl Sulfoxide
Dimethyl sulfoxide (DMSO) is an organosulfur compound with the formula ( CH3)2. This colorless liquid is the sulfoxide most widely used commercially. It is an important polar aprotic solvent that dissolves both polar and nonpolar compounds and is miscible in a wide range of organic solvents as well as water. It has a relatively high boiling point. DMSO has the unusual property that many individuals perceive a garlic-like taste in the mouth after DMSO makes contact with their skin. In terms of chemical structure, the molecule has idealized Cs symmetry. It has a trigonal pyramidal molecular geometry consistent with other three-coordinate S(IV) compounds, with a nonbonded electron pair on the approximately tetrahedral sulfur atom. Synthesis and production Dimethyl sulfoxide was first synthesized in 1866 by the Russian scientist Alexander Zaytsev, who reported his findings in 1867. Dimethyl sulfoxide is produced industrially from dimethyl sulfide, a by-product of the Kraf ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Anaphylaxis
Anaphylaxis is a serious, potentially fatal allergic reaction and medical emergency that is rapid in onset and requires immediate medical attention regardless of use of emergency medication on site. It typically causes more than one of the following: an itchy rash, throat closing due to swelling which can obstruct or stop breathing; severe tongue swelling which can also interfere with or stop breathing; shortness of breath, vomiting, lightheadedness, loss of consciousness, low blood pressure, and medical shock. These symptoms typically start in minutes to hours and then increase very rapidly to life-threatening levels. Urgent medical treatment is required to prevent serious harm or death, even if the patient has used an epipen or has taken other medications in response, and even if symptoms appear to be improving. Common causes include allergies to insect bites and stings, allergies to foods – including nuts, milk, fish, shellfish, eggs and some fresh fruits or dried fruits; a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pancytopenia
Pancytopenia is a medical condition in which there is significant reduction in the number of almost all blood cells (red blood cells, white blood cells, platelets, monocytes, lymphocytes, etc.). If only two parameters from the complete blood count are low, the term bicytopenia can be used. The diagnostic approach is the same as for pancytopenia. Causes Iatrogenic causes of pancytopenia include chemotherapy for malignancies if the drug or drugs used cause bone marrow suppression. Rarely, drugs (antibiotics, blood pressure medication, heart medication) can cause pancytopenia. For example, the antibiotic chloramphenicol can cause pancytopenia in some individuals. Rarely, pancytopenia may have other causes, such as mononucleosis or other viral diseases. Increasingly, HIV is itself a cause of pancytopenia. * Familial hemophagocytic syndrome * Aplastic anemia * Gaucher's disease * Metastatic carcinoma of bone * Multiple Myeloma * Overwhelming infections * Lymphoma * Myelofibrosi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cytopenia
Cytopenia is a reduction in the number of mature blood cells. It is common in cancer patients being treated with radiation and/or chemotherapy. Types Anemia – a reduction of the red blood cells in the body. Leukopenia – a deficiency of white blood cells, or leukocytes Neutropenia – a type of leukopenia, with a specific deficiency in neutrophils Thrombocytopenia – a deficiency of platelets Pancytopenia – when all three types of blood cells; red blood cells, white blood cells, and platelets, are all deficient. This is a life-threatening disorder that is a characteristic of aplastic anemia. There are also two general types of cytopenia: autoimmune and refractory. Autoimmune cytopenia – caused by an autoimmune disease when your body produces antibodies to destroy the healthy blood cells. Refractory cytopenia – caused by bone marrow not producing healthy blood cells, and can be a result of cancer. Symptoms and signs The symptoms of cytopenia vary depending on wh ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bloodstream Infections
Bloodstream infections (BSIs), which include bacteremias when the infections are bacterial and fungemias when the infections are fungal, are infections present in the blood. Blood is normally a sterile environment, so the detection of microbes in the blood (most commonly accomplished by blood cultures) is always abnormal. A bloodstream infection is different from sepsis, which is the host response to bacteria. Bacteria can enter the bloodstream as a severe complication of infections (like pneumonia or meningitis), during surgery (especially when involving mucous membranes such as the gastrointestinal tract), or due to catheters and other foreign bodies entering the arteries or veins (including during intravenous drug abuse). Transient bacteremia can result after dental procedures or brushing of teeth. Bacteremia can have several important health consequences. The immune response to the bacteria can cause sepsis and septic shock, which has a high mortality rate. Bacteria can a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Candidiasis
Candidiasis is a fungal infection due to any type of '' Candida'' (a type of yeast). When it affects the mouth, in some countries it is commonly called thrush. Signs and symptoms include white patches on the tongue or other areas of the mouth and throat. Other symptoms may include soreness and problems swallowing. When it affects the vagina, it may be referred to as a yeast infection or thrush. Signs and symptoms include genital itching, burning, and sometimes a white "cottage cheese-like" discharge from the vagina. Yeast infections of the penis are less common and typically present with an itchy rash. Very rarely, yeast infections may become invasive, spreading to other parts of the body. This may result in fevers along with other symptoms depending on the parts involved. More than 20 types of ''Candida'' can cause infection with ''Candida albicans'' being the most common. Infections of the mouth are most common among children less than one month old, the elderly, and those ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cytomegalovirus
''Cytomegalovirus'' (''CMV'') (from ''cyto-'' 'cell' via Greek - 'container' + 'big, megalo-' + -''virus'' via Latin 'poison') is a genus of viruses in the order ''Herpesvirales'', in the family ''Herpesviridae'', in the subfamily ''Betaherpesvirinae''. Humans and other primates serve as natural hosts. The 11 species in this genus include ''human betaherpesvirus 5'' (HCMV, human cytomegalovirus, HHV-5), which is the species that infects humans. Diseases associated with HHV-5 include mononucleosis and pneumonia. In the medical literature, most mentions of CMV without further specification refer implicitly to human CMV. Human CMV is the most studied of all cytomegaloviruses. MX2/MXB was identified as a restriction factor for herpesviruses, which acts at a very early stage of the replication cycle and MX2/MXB restriction of herpesvirus requires GTPase activity. Taxonomy Within the ''Herpesviridae'', CMV belongs to the ''Betaherpesvirinae'' subfamily, which also includes the g ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Complete Blood Count
A complete blood count (CBC), also known as a full blood count (FBC), is a set of medical laboratory tests that provide cytometry, information about the cells in a person's blood. The CBC indicates the counts of white blood cells, red blood cells and platelets, the concentration of hemoglobin, and the hematocrit (the volume percentage of red blood cells). The red blood cell indices, which indicate the average size and hemoglobin content of red blood cells, are also reported, and a white blood cell differential, which counts the different types of white blood cells, may be included. The CBC is often carried out as part of a medical assessment and can be used to monitor health or diagnose diseases. The results are interpreted by comparing them to Reference ranges for blood tests, reference ranges, which vary with sex and age. Conditions like anemia and thrombocytopenia are defined by abnormal complete blood count results. The red blood cell indices can provide information about the ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oncogene
An oncogene is a gene that has the potential to cause cancer. In tumor cells, these genes are often mutated, or expressed at high levels.Kimball's Biology Pages. "Oncogenes" Free full text Most normal cells will undergo a programmed form of rapid cell death () when critical functions are altered and malfunctioning. Activated oncogenes can cause those cells designated for apoptosis to survive and proliferate instead. Most oncogenes began as proto-oncogenes: normal genes involved in cell growth and proliferation or inhibition of apoptosis. If, through mutation, normal genes promoting cellular growth are up-regulated (gain-of-function mutation), they will predisp ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
