Rare diseases

Back in 1878 lung cancer was almost a rare disease; it was only one percent of all the different types of cancer. Scientists thought, in later years once the idea that cells split, reformed, and split again, that the reason there was little talk of cancer in the ancient world was because people did not live long enough. But in the EMPEROR OF ALL MALADIES by Siddhartha Mukherjee traces the history of the disease from 2500BCE to the present. By the early 1900s cancer seemed to be increasing. Or were people really just living long enough to get the illness diagnosed. The symptoms, diagnosis and treatment of lung cancer have been studied and analyze to have a better understanding of the disease, there is no cure yet but hopefully it’s in the near…

immoral business tactic used by pharmaceutical companies to generate large revenues involves the slight modification of an existing compound and using the new compound in the treatment of an orphan disease. In 2009 the company BioMarin Pharmaceutical created the drug Firdapse. The active substance is amifampridine and is used to treat the orphan disease, Lambert-Eaton myasthenic syndrome. In 2012 it was revealed that the product was simply a slight modification of 3,4 diaminopyridine, an…

Amicus Therapeutics is a biopharmaceutical company based in Cranbury, New Jersey. The company’s focus is on finding advanced therapies and treatments for a broad range of human genetic diseases. These diseases, known as orphan diseases, typically affect fewer than 200,000 people nationwide. Currently the company’s main concentration is on treatments for Lysosomal Storage Disorders such as Fabry disease and Pompe disease. Amicus Therapeutics has directed their development on the…

Angelman Syndrome Description of the Disease An extremely rare and uncommon disease known as Angelman Syndrome is a very complex neurodevelopmental and monogenic disorder (Bailus, 2014). The term neurodevelopmental refers to the shape, reshape, state and generation of the nervous system during the embryonic stages of life, while the term monogenic can be explained as a disease, which is controlled and inherited, by a single pair of genes (Gentile, 2009). It is estimated that Angelman Syndrome…

The most recent diagnostic criteria by Cohen et al. includes a number of specific benign tumors and malformations common with the condition. Some key specific criteria include cerebriform connective tissue nevi, hemihyperplasia with bone deformity , and dysregulated adipose and vascular tissue (Figure 3). The tumors caused by the disease are largely benign, and include lipomas, ovarian cystadenomas, monomorphic parotid adenomas , and meningiomas, though many other tumors benign and malignant…

company to gain share in the global market while keeping its core values unchanged. The focus of the company from the beginning has been putting patients first and the focus on the “two-price policy” – full price or free for patients in need and who could not afford it. HAND was created to help an untapped market, where neglected diseases such as tuberculosis kill as much as 2 million people a year in…

conditions that are perceived as solvable whereas conditions are external events that we encounter in our everyday lives. It is important for political actors to correctly identify problems and their causes so that the existing understandings of an issue can be challenged. For instance in the Medicare Catastrophic Coverage case, gaps in Medicare coverage left elderly Americans vulnerable to catastrophic illness. However, political actors realized the flaws in Medicare coverage and decided…

The Saint Louis Zoo The Saint Louis Zoo was voted second best zoo in the United States in a USA Today poll where they had the people who go to these zoos vote. The zoo is home to over 15,000 animals per their website. Many of them are rare and endangered species. They have some exhibits that they charge for but admission to the zoo is free. The Mission of the Saint Louis Zoo is to conserve animals and their habitats through animal management, research, recreation, and educational programs…

adding on to our $19.2 trillion debt. Also, immigrants may still be able to find a way around the wall, and those that are currently in America have no feasible solution to get out. Nevertheless, the economy will never get a chance to recover and improve its financial state with the addition of the wall. Habitats and ecosystems along the border will be broken up, and could be possibly destroyed if the plan of building a wall goes through. Several rare and endangered species inhibit the…

Rare earths are very significant when it comes to the development and use of technical equipment as they can be very important to the performance of the unit. An example of an everyday piece of technology that uses rare earths is the IPhone. Apple has said that the IPhone uses eight rare earths, from everything from the colored screen, to speakers, to the miniature circuits inside the phone. Although the amount of rare earths in the phone is very little, the demand for rare earths will continue…