...Idiopathic Thrombocytopenic Pupura (ITP) is a disease that targets and destroys platelets. No one is sure how this disease is contracted because the etiology is unclear. Some doctors can, however, guess how the patient contracted the disease depending on certain factors. Most of the time, they are unlikely to figure out the cause of the disease. This disease can cause someone to bleed to death if they were to get a cut because they wouldn’t have something to clot the blood. ITP does not have a cure or a treatment that works for all patients. Patients of all demographics have reacted differently to various treatments to the disease. Patients and guardians of the patient also have an ability to choose what type of treatment they would like to...
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...JUVENILE RHEUMATOID ARTHRITIS Kevin Weller Kaplan University College Composition II CM220 Prof: Diana Gerow August 26, 2012 JUVENILE RHEUMATOID ARTHRITIS There is currently no known cause and no cure for Juvenile Arthritis, so it is my goal to raise awareness for those in the community that are not aware that children can get arthritis. Many people believe that arthritis only affects older people or those who have worked hard and abused their body over many years. Children still need support to remain active to try and beat the pain and stiffness they are dealing with daily and the community is also important to help support the efforts to find a cure and help those currently impacted with the disease. Juvenile arthritis affects children under the age of 16. JRA can show up overnight and blood tests cannot determine if a child has arthritis. There is currently no cause or cure for this disease that affects 50,000 children in the United States. JRA is often misunderstood or mistaken for other conditions, and thus parents need to learn the signs of Juvenile Rheumatoid Arthritis, how to find appropriate medical intervention, and what best practices they can use at home. Juvenile Arthritis is an inflammatory condition that develops in children ages 16 years and younger. Juvenile Rheumatoid Arthritis is the most common type and there is no known cause although hereditary seems to play a part. Arthritis affects approximately 1 out of every 100,000 children mildly and...
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...Orphan Drug Report An orphan drug is a drug designed to treat a rare disease or condition. For a drug to qualify for orphan status both the drug and disease or condition must meet certain criteria specified in the Orphan Drug Act (ODA). This designation would qualify the sponsor of the drug for developmental incentives which includes waiving of regulator fees, faster or simpler clinical trial and approval process, tax credit or grants to offset research and development, and a period of marketing exclusivity which means no competition for many years. it does not mean that the FDA is less strict when it comes to safety and effectiveness. All of this is specified within the FDA’s implementing regulations at 21 CFR Part 316. It’s important for these incentives to be available because there may only be a few thousand who suffer from a rare condition or disease. For the pharmaceutical industry to develop any drug it must see a certain amount of profit potential. Most of these disease are genetic, many appear early in life, and 30 percent of children with orphan diseases die before age of five. Although the market is small for orphan drugs, there has been notable growth in recent years. Currently, 350 orphan drugs are approved for sale in the U.S. A good example is Rituxan from Genentech, the second most profitable drug in the world, given orphan status to treat B-cell Non-Hogkin’s lymphoma. In 2010, it yielded $5.24 billion in sales for its use as an orphan drug...
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...Disease Achondroplasia is the most frequent form of short-limb dwarfism (Nahar R et al 2009). As well as short stature due to shortening of limbs, affected individuals have characteristic facies with frontal bossing and mid-face hypoplasia, exaggerated lumbar lordosis, limitation of elbow extension, genu varum and trident-like hands. Incidence increases with paternal age. Achondroplasia is caused, in virtually all of the cases, by a G380R mutation in fibroblast growth factor receptor 3 (FGFR3) (Di Rocco F et al 2014). FGFR3 is also important in craniofacial, vertebral and neurological development such that this mutation has multiple effects in an affected individual (Horton WA et al 2007). Clinical Presentation Gross motor development frequently is delayed. Motor milestones such as head control and independent sitting, standing, and ambulation may lag by 3-6 months. Speech and language problems may be caused by tongue thrust (due to abnormal maxillomandibular relationship) but often resolve spontaneously. Twenty percent of patients experience delayed speech acquisition. Cognitive skills are preserved, and the intelligence level is within normal limits. Cranial enlargement and poor head control place the infant at risk for extension injuries. An Australian study assessed the functional milestones of achondroplasia children aged 3-7 years. The data noted that while milestones were delayed across all ages studied, functioning improved between age 3 and 5 years, although not...
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...There are many different treatments for Idiopathic thrombocytopenic purpura that depends on the severity of the patient, the age of the patient, and the type of Idiopathic thrombocytopenic purpura the patient has acute or chronic. Children with this disease don’t need much treatment, in most cases children with ITP will improve without any treatment. Close monitoring of the child and keeping them from any serious bleeding, until the body had a chance to correct the disorder by its self is what is recommended for children. The Children’s Hospital of Philadelphia says that “many children with Idiopathic thrombocytopenic purpura are able to spontaneously recover in about 2 to 4 days” (Children’s Hospital). If the child does need treatment the one doctor’s go to first is to put the child on a steroid which...
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...1. How to care for the child with a chronic condition or special needs. Give child choices, some sort of control/independence. Treat them like a normal kid. Mainstreaming: In school in a regular classroom. Do not separate these kids and make them feel different. Don’t focus on things they cannot do but rather focus on what they could do. . Use a more positive approach. Don’t focus on their chronological age because two six year olds will be different. Overall integration into society without stigmas. 2. Phases of reactions that parents go through related to a child with a chronic illness or disability 5 stages 1) Shock 2) Denial 3) Anger 4) Guilt 5) Acceptance. These 5 steps can happen over and over again. i.e., it’s prom night or communion and their child is not like all the other children and the parent may go through these steps again. 3. What is chronic sorrow? A parent dealing with a child with a chronic condition goes through the adjustment process and grief experience it does not happen in one experience. It may happen over and over again when different situations come up. Recurrent adjustment. 4. How to foster an environment of normalcy. Letting them go to a regular school, mainstreaming them, giving them control, treating them overall like a kid, not focusing on chronological age, do not compare to other kids in their age group. 5. Iron deficiency anemia Why does a child get it? We do not see it in the beginning with babies because they have iron stores...
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...Thrombotic Thrombocytopenic Purpura, also acknowledged as TTP, is a rare and serious blood disorder represented by hemolytic anemia and thrombocytopenia coinciding with renal failure and neurological dysfunction (Onundarson, Heal, Rowe, Apr. 1992). The exact reasoning for hemolytic anemia is vague but it may be a consequence of the motorized disintegration of erythrocytes as they pour through partially occluded micro vessels (Onundarson, Heal, Rowe, 1992). TTP is known to cause red blood cells to break apart faster than the body can replace them, conducting hemolytic anemia which is a rare form of anemia. In TTP, small blood vessels begin to clot throughout the body. “These clots can obstruct the flow of oxygen-rich blood to the body’s organs, such as the brain, kidneys and heart” (NHLB, 2014). As a result, serious health problems develop. This increased clotting in TTP also uses up platelets in the blood. These platelets set apart damaged blood vessels, unite together to close up small cuts and cease the bleeding. Stagnation in the ADAMTS13 enzyme, a type of protein in the blood, plays a key role in “acquired” TTP and was discovered in TTP patients. The ADAMTS13 gene regulates the enzyme, which is involved in blood clotting. “The enzyme breaks up a large protein called von Willebrand factor that clumps together with platelets to form blood clots.” (Onundarson, Heal, Rowe, Apr. 1992). A strong platelet count ranges from about 150,000 to 450,000 per micro liter of blood...
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